James Wilson | Academic Influence

Q: What Schools Are Affiliated With James Wilson

James Wilson is affiliated with the following schools: Jikei University School of Medicine, KU Leuven, University of Michigan, University of California, Los Angeles, University of Notre Dame, Villanova University, Albion College, University of Pennsylvania

Why Is James Wilson Influential?

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According to Wikipedia, James M. Wilson is a biomedical researcher with expertise in gene therapy. Wilson graduated from Albion College and the University of Michigan . He completed residency training in Internal Medicine at the Massachusetts General Hospital followed by a postdoctoral fellowship at the Whitehead Institute.

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James Wilson 's Published Works

Number of citations in a given year to any of this author's works

Total number of citations to an author for the works they published in a given year. This highlights publication of the most important work(s) by the author

Published Works

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy (2002) (1443)
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. (2003) (1388)
Human β-Defensin-1 Is a Salt-Sensitive Antibiotic in Lung That Is Inactivated in Cystic Fibrosis (1997) (1118)
Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues (2004) (922)
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics (2008) (687)
Submucosal glands are the predominant site of CFTR expression in the human bronchus (1992) (681)
Recombinant adeno-associated virus for muscle directed gene therapy (1997) (678)
Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. (2009) (624)
A model system for in vivo gene transfer into the central nervous system using an adenoviral vector (1993) (590)
Gene Therapy Vectors Based on Adeno-Associated Virus Type 1 (1999) (558)
Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia (1994) (555)
Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer (1990) (554)
A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia (1995) (526)
Transduction of Dendritic Cells by DNA Viral Vectors Directs the Immune Response to Transgene Products in Muscle Fibers (1998) (489)
CD40 Ligand-Dependent T Cell Activation: Requirement of B7-CD28 Signaling Through CD40 (1996) (489)
New recombinant serotypes of AAV vectors. (2005) (471)
Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis (1994) (459)
A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice (2016) (434)
Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humans (1994) (369)
Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery (2002) (367)
A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency. (2002) (358)
Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial (2015) (353)
Natural Killer T Cell Ligand α-Galactosylceramide Enhances Protective Immunity Induced by Malaria Vaccines (2002) (328)
Augmentation of Innate Host Defense by Expression of a Cathelicidin Antimicrobial Peptide (1999) (326)
Adeno-associated viruses undergo substantial evolution in primates during natural infections (2003) (325)
Direct gene transfer of human CFTR into human bronchial epithelia of xenografts with E1–deleted adenoviruses (1993) (310)
Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo (2001) (309)
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results. (2011) (305)
Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. (2007) (304)
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy (2009) (303)
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN. (2018) (278)
Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. (2010) (270)
Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. (2005) (263)
Recombinant IL–12 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repeated gene therapy to mouse lung (1995) (262)
HIV-1 suppression and durable control by combining single broadly neutralizing antibodies and antiretroviral drugs in humanized mice (2013) (255)
Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat. (2008) (251)
Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency. (2009) (250)
The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice. (2018) (245)
β-Defensin 1 Contributes to Pulmonary Innate Immunity in Mice (2002) (235)
Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents (2011) (229)
“Stealth” Adenoviruses Blunt Cell-Mediated and Humoral Immune Responses against the Virus and Allow for Significant Gene Expression upon Readministration in the Lung (2001) (229)
A Simian Replication-Defective Adenoviral Recombinant Vaccine to HIV-1 Gag 1 (2003) (228)
Novel Adeno-Associated Virus Serotypes Efficiently Transduce Murine Photoreceptors (2007) (223)
Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector (1999) (223)
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid (2006) (215)
Human Airway Epithelial Cells Sense Pseudomonas aeruginosa Infection via Recognition of Flagellin by Toll-Like Receptor 5 (2005) (213)
Hybrid Vectors Based on Adeno-Associated Virus Serotypes 2 and 5 for Muscle-Directed Gene Transfer (2001) (212)
Humoral Immunity to Adeno-Associated Virus Type 2 Vectors following Administration to Murine and Nonhuman Primate Muscle (2000) (209)
Mouse β-Defensin 1 Is a Salt-Sensitive Antimicrobial Peptide Present in Epithelia of the Lung and Urogenital Tract (1998) (208)
Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteins. (2002) (207)
Novel, Chimpanzee Serotype 68-Based Adenoviral Vaccine Carrier for Induction of Antibodies to a Transgene Product (2002) (206)
Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver (2009) (203)
Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. (2004) (203)
Mouse β-Defensin 3 Is an Inducible Antimicrobial Peptide Expressed in the Epithelia of Multiple Organs (1999) (194)
Novel AAV serotypes for improved ocular gene transfer (2008) (187)
Bacterial Phosphorylcholine Decreases Susceptibility to the Antimicrobial Peptide LL-37/hCAP18 Expressed in the Upper Respiratory Tract (2000) (185)
Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus–mediated transfer of the VLDL receptor gene (1996) (182)
Humoral Immune Response to AAV (2013) (180)
Lessons learned from the clinical development and market authorization of Glybera. (2013) (180)
Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey (2011) (180)
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN (2018) (177)
Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. (2005) (175)
PEGylation of E1-deleted adenovirus vectors allows significant gene expression on readministration to liver. (2002) (175)
Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. (2008) (174)
Adeno-Associated Virus as a Vector for Liver-Directed Gene Therapy (1998) (169)
The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice. (2011) (166)
CpG-depleted adeno-associated virus vectors evade immune detection. (2013) (161)
Prolonged Metabolic Correction in Adult Ornithine Transcarbamylase-deficient Mice with Adenoviral Vectors (*) (1996) (160)
Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors. (2011) (157)
Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. (2006) (156)
Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector. (2007) (156)
Longitudinal evaluation and assessment of cardiovascular disease in patients with homozygous familial hypercholesterolemia. (2008) (155)
Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered (2006) (152)
Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV (1999) (151)
Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. (2002) (151)
Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. (2009) (149)
In vivo somatic cell gene transfer of an engineered Noggin mutein prevents BMP4-induced heterotopic ossification. (2003) (148)
PEGylation of a Vesicular Stomatitis Virus G Pseudotyped Lentivirus Vector Prevents Inactivation in Serum (2004) (143)
Human cone photoreceptor dependence on RPE65 isomerase (2007) (142)
Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents. (2002) (141)
Universal protection against influenza infection by a multidomain antibody to influenza hemagglutinin (2018) (141)
Chimpanzee adenovirus vaccine protects against Zaire Ebola virus. (2006) (140)
Sustained Production of β-Glucuronidase from Localized Sites after AAV Vector Gene Transfer Results in Widespread Distribution of Enzyme and Reversal of Lysosomal Storage Lesions in a Large Volume of Brain in Mucopolysaccharidosis VII Mice (1999) (139)
Erythropoietin gene therapy leads to autoimmune anemia in macaques. (2004) (139)
Isolation and Characterization of Adenoviruses Persistently Shed from the Gastrointestinal Tract of Non-Human Primates (2009) (138)
Effect of blood collection technique in mice on clinical pathology parameters. (2002) (136)
Toll-Like Receptor 4 Mediates Innate Immune Responses to Haemophilus influenzae Infection in Mouse Lung1 (2002) (135)
Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. (2006) (134)
Resolution of Primary Severe Acute Respiratory Syndrome-Associated Coronavirus Infection Requires Stat1 (2004) (131)
Cytotoxic T-Lymphocyte Target Proteins and Their Major Histocompatibility Complex Class I Restriction in Response to Adenovirus Vectors Delivered to Mouse Liver (1998) (129)
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. (2013) (127)
Induction of CD8+ T Cells to an HIV-1 Antigen through a Prime Boost Regimen with Heterologous E1-Deleted Adenoviral Vaccine Carriers 1 (2003) (125)
Macaque Model for Severe Acute Respiratory Syndrome (2004) (125)
AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice. (2004) (124)
High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses. (2006) (123)
Long-term Follow-up of Patients with Malignant Pleural Mesothelioma Receiving High-Dose Adenovirus Herpes Simplex Thymidine Kinase/Ganciclovir Suicide Gene Therapy (2005) (123)
Physiological modulation of CFTR activity by AMP-activated protein kinase in polarized T84 cells. (2003) (122)
The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier. (2019) (121)
Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. (2010) (120)
Fas ligand—a double-edged sword (1998) (119)
The complex and evolving story of T cell activation to AAV vector-encoded transgene products. (2011) (118)
The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. (2010) (117)
Lentiviral vectors pseudotyped with minimal filovirus envelopes increased gene transfer in murine lung. (2003) (117)
Gene transfer into skeletal muscle using novel AAV serotypes (2005) (114)
Structure-Based Identification of a Major Neutralizing Site in an Adenovirus Hexon (2006) (113)
Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets. (2007) (112)
No evidence for tumorigenesis of AAV vectors in a large-scale study in mice. (2005) (110)
Gene Therapy for Cystic Fibrosis Using E1-Deleted Adenovirus: A Phase I Trial in the Nasal Cavity. University of North Carolina at Chapel Hill, Chapel Hill, North Carolina (1994) (109)
Moving forward after two deaths in a gene therapy trial of myotubular myopathy. (2020) (107)
The genesis of cystic fibrosis lung disease (106)
Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model. (2011) (105)
Treatment of Experimental Human Mesothelioma Using Adenovirus Transfer of the Herpes Simplex Thymidine Kinase Gene (1995) (105)
Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing. (2010) (103)
A History Lesson for Stem Cells (2009) (102)
Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes. (2002) (101)
Absolute determination of single-stranded and self-complementary adeno-associated viral vector genome titers by droplet digital PCR. (2014) (97)
Identification of the Galactose Binding Domain of the Adeno-Associated Virus Serotype 9 Capsid (2012) (97)
Airway epithelia regulate expression of human β‐defensin 2 through toll‐like receptor 2 (2003) (97)
Toll-Like Receptor 4 Mediates Innate Immune Responses to Haemophilus influenzae Infection in Mouse Lung (2001) (95)
Intranasal Antibody Gene Transfer in Mice and Ferrets Elicits Broad Protection Against Pandemic Influenza (2013) (94)
Gendicine: The First Commercial Gene Therapy Product; Chinese Translation of Editorial (2005) (94)
Gene therapy in a xenograft model of cystic fibrosis lung corrects chloride transport more effectively than the sodium defect (1995) (93)
Gene therapy with novel adeno‐associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia (2004) (93)
Characterization of a family of chimpanzee adenoviruses and development of molecular clones for gene transfer vectors. (2004) (92)
Pharmacological regulation of protein expression from adeno-associated viral vectors in the eye. (2002) (90)
Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I. (2014) (89)
Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector. (2012) (89)
Partial protection against H5N1 influenza in mice with a single dose of a chimpanzee adenovirus vector expressing nucleoprotein. (2007) (88)
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology. (2020) (83)
Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping. (2012) (83)
AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina (2013) (83)
Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids. (2015) (83)
Human cystic fibrosis transmembrane conductance regulator directed to respiratory epithelial cells of transgenic mice (1992) (81)
Preexisting Immunity to Adenovirus in Rhesus Monkeys Fails To Prevent Vector-Induced Toxicity (2002) (81)
Adenovirus-mediated correction of the genetic defect in hepatocytes from patients with familial hypercholesterolemia (1993) (81)
AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta). (2011) (80)
Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. (2009) (79)
High-Level Transgene Expression in Nonhuman Primate Liver with Novel Adeno-Associated Virus Serotypes Containing Self-Complementary Genomes (2006) (78)
Role of E4 in Eliciting CD4 T-Cell and B-Cell Responses to Adenovirus Vectors Delivered to Murine and Nonhuman Primate Lungs (1998) (77)
Complete Deficiency of the Low-Density Lipoprotein Receptor Is Associated With Increased Apolipoprotein B-100 Production (2005) (77)
Specific AAV serotypes stably transduce primary hippocampal and cortical cultures with high efficiency and low toxicity (2008) (76)
Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol (2018) (73)
Ex Vivo Gene Therapy of Familial Hypercholesterolemia. Howard Hughes Medical Institute, University of Michigan (1992) (73)
β-Defensin 1 Plays a Role in Acute Mucosal Defense against Candida albicans (2015) (73)
Transduction of Well-Differentiated Airway Epithelium by Recombinant Adeno-Associated Virus Is Limited by Vector Entry (1999) (73)
Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors. (2006) (73)
Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals. (2018) (72)
Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna (2014) (72)
Gene transfer of wild-type apoA-I and apoA-I Milano reduce atherosclerosis to a similar extent (2007) (71)
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency (2017) (70)
Host Immune Responses to Chronic Adenovirus Infections in Human and Nonhuman Primates (2008) (70)
A Common Mechanism for Cytoplasmic Dynein-Dependent Microtubule Binding Shared among Adeno-Associated Virus and Adenovirus Serotypes (2006) (70)
Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates. (2015) (70)
Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints (2009) (69)
Gene Therapy in a Humanized Mouse Model of Familial Hypercholesterolemia Leads to Marked Regression of Atherosclerosis (2010) (69)
AAV as an immunogen. (2007) (69)
Adeno-Associated Virus Capsid Structure Drives CD4-Dependent CD8+ T Cell Response to Vector Encoded Proteins1 (2009) (68)
Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening. (2019) (68)
Identification of murine CD8 T cell epitopes in codon-optimized SARS-associated coronavirus spike protein (2005) (68)
Chimpanzee adenovirus CV-68 adapted as a gene delivery vector interacts with the coxsackievirus and adenovirus receptor. (2002) (68)
MR and fluorescent imaging of low-density lipoprotein receptors. (2004) (68)
Rep/Cap gene amplification and high-yield production of AAV in an A549 cell line expressing Rep/Cap. (2002) (66)
Analysis of particle content of recombinant adeno-associated virus serotype 8 vectors by ion-exchange chromatography. (2012) (66)
Utility of PEGylated recombinant adeno-associated viruses for gene transfer. (2005) (66)
Adeno-associated virus serotype 8 gene therapy leads to significant lowering of plasma cholesterol levels in humanized mouse models of homozygous and heterozygous familial hypercholesterolemia. (2013) (65)
Adenovirus-based vaccine prevents pneumonia in ferrets challenged with the SARS coronavirus and stimulates robust immune responses in macaques (2007) (64)
Nonhuman primate models for diabetic ocular neovascularization using AAV2-mediated overexpression of vascular endothelial growth factor. (2005) (63)
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII (2015) (63)
Vaccines Based on Novel Adeno-Associated Virus Vectors Elicit Aberrant CD8+ T-Cell Responses in Mice (2007) (62)
Inverse zonation of hepatocyte transduction with AAV vectors between mice and non-human primates. (2011) (62)
Proteasome inhibition enhances AAV-mediated transgene expression in human synoviocytes in vitro and in vivo. (2005) (62)
Scalable mRNA and siRNA Lipid Nanoparticle Production Using a Parallelized Microfluidic Device. (2021) (62)
Correction of the dystrophic phenotype by in vivo targeting of muscle progenitor cells. (2003) (61)
Readministration of Adenovirus Vector in Nonhuman Primate Lungs by Blockade of CD40-CD40 Ligand Interactions (2000) (61)
Adenoviruses in Lymphocytes of the Human Gastro-Intestinal Tract (2011) (61)
The special case of gene therapy pricing (2014) (61)
A new scalable method for the purification of recombinant adenovirus vectors. (2002) (60)
Effect of Preexisting Immunity on an Adenovirus Vaccine Vector: In Vitro Neutralization Assays Fail To Predict Inhibition by Antiviral Antibody In Vivo (2009) (60)
A New Genetic Vaccine Platform Based on an Adeno-Associated Virus Isolated from a Rhesus Macaque (2009) (59)
Diet-dependent cardiovascular lipid metabolism controlled by hepatic LXRalpha. (2005) (59)
RPGR-associated retinal degeneration in human X-linked RP and a murine model. (2012) (59)
Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 achieves global cardiac gene transfer in canines. (2008) (59)
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates (2020) (59)
Adenovirus-based genetic vaccines for biodefense. (2005) (58)
Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques (2018) (58)
Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway. (2010) (57)
Gendicine: the first commercial gene therapy product. (2005) (56)
Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates. (2005) (56)
Mucosal delivery of adenovirus-based vaccine protects against Ebola virus infection in mice. (2007) (55)
Efficacy of severe acute respiratory syndrome vaccine based on a nonhuman primate adenovirus in the presence of immunity against human adenovirus. (2006) (55)
Stat1-dependent induction of tumor necrosis factor-related apoptosis-inducing ligand and the cell-surface death signaling pathway by interferon beta in human cancer cells. (2003) (54)
Regulated expression of erythropoietin from an AAV vector safely improves the anemia of beta-thalassemia in a mouse model. (2003) (53)
Rhesus Monkey (Macaca mulatta) Mucosal Antimicrobial Peptides Are Close Homologues of Human Molecules (2001) (53)
Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing. (2009) (52)
Stroma formation and angiogenesis by overexpression of growth factors, cytokines, and proteolytic enzymes in human skin grafted to SCID mice. (2003) (51)
Human gene marker/therapy clinical protocols. (1999) (51)
Generation of an adenoviral vaccine vector based on simian adenovirus 21. (2006) (51)
In vivo detection of gene expression in liver by 31P nuclear magnetic resonance spectroscopy employing creatine kinase as a marker gene (2001) (50)
Induction of Protective Immunity to Anthrax Lethal Toxin with a Nonhuman Primate Adenovirus-Based Vaccine in the Presence of Preexisting Anti-Human Adenovirus Immunity (2005) (50)
Delivery of Adeno-Associated Virus Vectors to the Fetal Retina: Impact of Viral Capsid Proteins on Retinal Neuronal Progenitor Transduction (2003) (49)
AAV vectors avoid inflammatory signals necessary to render transduced hepatocyte targets for destructive T cells. (2010) (49)
Cell Transplantation in Liver-Directed Gene Therapy (1993) (49)
Complete Prevention of Atherosclerosis in ApoE-Deficient Mice by Hepatic Human ApoE Gene Transfer With Adeno-Associated Virus Serotypes 7 and 8 (2006) (49)
Transduction of human islets with pseudotyped lentiviral vectors. (2004) (49)
Vehicles for gene therapy (1993) (49)
Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model (2015) (48)
Complete nucleotide sequences and genome organization of four chimpanzee adenoviruses. (2004) (48)
AAV Vectors Expressing LDLR Gain-of-Function Variants Demonstrate Increased Efficacy in Mouse Models of Familial Hypercholesterolemia (2014) (47)
Systemic IFN-β gene therapy results in long-term survival in mice with established colorectal liver metastases (2001) (47)
Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome. (2012) (47)
Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques (2018) (46)
Targeted delivery of antisense oligonucleotides by molecular conjugates (1992) (46)
A pilot study of systemic corticosteroid administration in conjunction with intrapleural adenoviral vector administration in patients with malignant pleural mesothelioma (2000) (46)
Deamidation of Amino Acids on the Surface of Adeno-Associated Virus Capsids Leads to Charge Heterogeneity and Altered Vector Function. (2018) (46)
AAV8 induces tolerance in murine muscle as a result of poor APC transduction, T cell exhaustion, and minimal MHCI upregulation on target cells. (2014) (46)
CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX knockout mice. (2019) (46)
Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines. (2011) (46)
Preexisting Neutralizing Antibodies to Adeno-Associated Virus Capsids in Large Animals Other Than Monkeys May Confound In Vivo Gene Therapy Studies. (2015) (46)
The Next Chapter. (2015) (45)
Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency (2017) (45)
The p38 Mitogen-Activated Protein Kinase Signaling Pathway Is Coupled to Toll-Like Receptor 5 To Mediate Gene Regulation in Response to Pseudomonas aeruginosa Infection in Human Airway Epithelial Cells (2007) (45)
Impact of preexisting vector immunity on the efficacy of adeno-associated virus-based HIV-1 Gag vaccines. (2008) (45)
Intramuscular Injection of AAV8 in Mice and Macaques Is Associated with Substantial Hepatic Targeting and Transgene Expression (2014) (43)
Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I (2014) (43)
MHC-independent presentation of mycobacteria to human γδ T cells (1993) (42)
Intravenous Injection of an Adenovirus Encoding Hepatocyte Growth Factor Results in Liver Growth and Has a Protective Effect Against Apoptosis (2000) (42)
An optimized protocol for detection of E. coli β-galactosidase in lung tissue following gene transfer (2005) (42)
Characterization of stem cells in human airway capable of reconstituting a fully differentiated bronchial epithelium (1995) (42)
Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys. (2019) (42)
Structure of neurotropic adeno-associated virus AAVrh.8. (2015) (41)
Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors (2005) (41)
Activation of Transgene-specific T Cells Following Lentivirus-mediated Gene Delivery to Mouse Lung (2009) (41)
Adenovirus-adeno-associated virus hybrid for large-scale recombinant adeno-associated virus production. (2009) (41)
Adenovirus-Mediated in Vivo Gene Transfer Rapidly Protects Ornithine Transcarbamylase-Deficient Mice from an Ammonium Challenge (1997) (40)
Use of chimeric adenoviral vectors to assess capsid neutralization determinants. (2004) (40)
Fetal gene therapy: Efficacy, toxicity, and immunologic effects of early gestation recombinant adenovirus (1999) (40)
Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs. (2011) (39)
Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction. (2013) (39)
An AAV-based, room-temperature-stable, single-dose COVID-19 vaccine provides durable immunogenicity and protection in non-human primates (2021) (39)
Sustained Correction of OTC Deficiency in Spf ash mice Using Optimized Self-complementary AAV2/8 Vectors (2011) (39)
Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/- Rhesus Macaques. (2017) (38)
High throughput creation of recombinant adenovirus vectors by direct cloning, green-white selection and I-Sce I-mediated rescue of circular adenovirus plasmids in 293 cells (2003) (38)
In vivo quantitative noninvasive imaging of gene transfer by single-photon emission computerized tomography. (2003) (38)
AAV Natural Infection Induces Broad Cross-Neutralizing Antibody Responses to Multiple AAV Serotypes in Chimpanzees. (2016) (37)
Creation of a panel of vectors based on ape adenovirus isolates (2011) (36)
Generation of a mouse expressing a conditional knockout of the hepatocyte growth factor gene: demonstration of impaired liver regeneration. (2004) (36)
Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8. (2013) (36)
Combination Therapy with Lamivudine and Adenovirus Causes Transient Suppression of Chronic Woodchuck Hepatitis Virus Infections (2000) (36)
Mucosally Delivered E1-Deleted Adenoviral Vaccine Carriers Induce Transgene Product-Specific Antibody Responses in Neonatal Mice 1 (2003) (36)
Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency (2017) (35)
Towards liver-directed gene therapy: Retrovirus-mediated gene transfer into human hepatocytes (1991) (35)
A mutation-independent CRISPR-Cas9–mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency (2020) (35)
An AAV Vector-Mediated Gene Delivery Approach Facilitates Reconstitution of Functional Human CD8+ T Cells in Mice (2014) (35)
A CD46-binding chimpanzee adenovirus vector as a vaccine carrier. (2007) (35)
Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia. (2013) (35)
Biology of E1-Deleted Adenovirus Vectors in Nonhuman Primate Muscle (2001) (35)
Regulatory and Exhausted T Cell Responses to AAV Capsid. (2017) (34)
Overexpression of Cyclin A Inhibits Augmentation of Recombinant Adeno-Associated Virus Transduction by the Adenovirus E4orf6 Protein (1999) (34)
Identification of an adeno-associated virus binding epitope for AVB sepharose affinity resin (2015) (33)
Immunogenicity in Mamu-A*01 rhesus macaques of a CCR5-tropic human immunodeficiency virus type 1 envelope from the primary isolate (Bx08) after synthetic DNA prime and recombinant adenovirus 5 boost. (2003) (33)
Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model. (2016) (33)
Inflammation promotes the loss of adeno-associated virus-mediated transgene expression in mouse liver. (2011) (33)
Intramuscular administration of AAV overcomes pre-existing neutralizing antibodies in rhesus macaques. (2016) (33)
AAV8-antiVEGFfab Ocular Gene Transfer for Neovascular Age-Related Macular Degeneration. (2017) (33)
Efficient gene transfer into the mouse lung by fetal intratracheal injection of rAAV2/6.2. (2010) (32)
Construction of adenoviral vectors (2001) (32)
Adeno-Associated Virus 9-Mediated Airway Expression of Antibody Protects Old and Immunodeficient Mice against Influenza Virus (2014) (31)
AAV natural infection induces broad cross-neutralizing antibody responses to multiple AAV serotypes in chimpanzees. (2016) (31)
Adenoviruses in Fecal Samples from Asymptomatic Rhesus Macaques, United States (2012) (31)
Efficient transduction of human monocyte-derived dendritic cells by chimpanzee-derived adenoviral vector. (2003) (31)
The structure of AAVrh32.33, a novel gene delivery vector. (2014) (31)
iNOS Expression In Dystrophinopathies Can Be Reduced By Somatic Gene Transfer of Dystrophin or Utrophin (2001) (31)
Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice. (2016) (31)
Enhancing the utility of adeno-associated virus gene transfer through inducible tissue-specific expression. (2013) (31)
Optical imaging of Ca2+‐evoked fluid secretion by murine nasal submucosal gland serous acinar cells (2007) (30)
Hepatocellular carcinoma in a research subject with ornithine transcarbamylase deficiency. (2012) (30)
Human fetal trachea-SCID mouse xenografts: efficacy of vesicular stomatitis virus-G pseudotyped lentiviral-mediated gene transfer. (2003) (30)
AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice. (2017) (30)
Adenovirus-based vaccines generate cytotoxic T lymphocytes to epitopes of NS1 from dengue virus that are present in all major serotypes. (2008) (30)
Human immune system mice immunized with Plasmodium falciparum circumsporozoite protein induce protective human humoral immunity against malaria. (2015) (29)
Molecular analysis of vector genome structures after liver transduction by conventional and self-complementary adeno-associated viral serotype vectors in murine and nonhuman primate models. (2010) (29)
BALB/c mice show impaired hepatic tolerogenic response following AAV gene transfer to the liver. (2010) (29)
Increased Mucosal CD4+ T Cell Activation in Rhesus Macaques following Vaccination with an Adenoviral Vector (2014) (29)
Adenovirus-mediated gene transfer to liver1 (2001) (28)
Creatine kinase, a magnetic resonance-detectable marker gene for quantification of liver-directed gene transfer. (2005) (28)
Helper lipid structure influences protein adsorption and delivery of lipid nanoparticles to spleen and liver. (2021) (27)
Assessment of Humoral, Innate, and T-Cell Immune Responses to Adeno-Associated Virus Vectors. (2018) (27)
Widespread dispersion of adeno-associated virus serotype 1 and adeno-associated virus serotype 6 vectors in the rat central nervous system and in human glioblastoma multiforme xenografts. (2005) (27)
Combined 5-Fluorouracil/Systemic Interferon-β Gene Therapy Results in Long-Term Survival in Mice with Established Colorectal Liver Metastases (2004) (27)
Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia. (2017) (27)
Human immunodeficiency viral vector pseudotyped with the spike envelope of severe acute respiratory syndrome coronavirus transduces human airway epithelial cells and dendritic cells. (2007) (26)
Targeting viral-mediated transduction to the lung airway epithelium with the anti-inflammatory cationic lipid dexamethasone-spermine. (2005) (26)
Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia. (2016) (26)
Long-term Stable Reduction of Low-density Lipoprotein in Nonhuman Primates Following In Vivo Genome Editing of PCSK9. (2021) (26)
Round two for liver gene therapy (1996) (25)
Innate Immune Responses to Adenoviral Vector-Mediated Acute Pancreatitis (2005) (24)
Regulated Gene Expression in Gene Therapy (2001) (24)
Th2-Dependent B Cell Responses in the Absence of CD40-CD40 Ligand Interactions1 (2000) (24)
Adenovirus-Antibody Complexes Contributed to Lethal Systemic Inflammation in a Gene Therapy Trial. (2020) (24)
Lung homing CTLs and their proliferation ability are important correlates of vaccine protection against influenza. (2010) (23)
Oral Vaccination of Mice with Adenoviral Vectors Is Not Impaired by Preexisting Immunity to the Vaccine Carrier (2005) (23)
Assessment of humoral, innate, and T-cell immune responses to adeno-associated virus vectors. (2018) (23)
Mapping the Structural Determinants Responsible for Enhanced T Cell Activation to the Immunogenic Adeno-Associated Virus Capsid from Isolate Rhesus 32.33 (2013) (23)
Adeno-associated virus and lentivirus pseudotypes for lung-directed gene therapy. (2004) (23)
A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator ( CFTR) gene therapy in patients with cystic fibrosis (2016) (23)
TLR9 signaling mediates adaptive immunity following systemic AAV gene therapy. (2019) (23)
Cationic lipid formulations alter the in vivo tropism of AAV2/9 vector in lung. (2009) (23)
Intranasal gene therapy to prevent infection by SARS-CoV-2 variants (2021) (23)
Mapping an Adeno-associated Virus 9-Specific Neutralizing Epitope To Develop Next-Generation Gene Delivery Vectors (2018) (22)
Vectored Expression of the Broadly Neutralizing Antibody FI6 in Mouse Airway Provides Partial Protection against a New Avian Influenza A Virus, H7N9 (2013) (22)
ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing (2020) (22)
Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration (2020) (22)
A Comparative Analysis of Novel Fluorescent Proteins as Reporters for Gene Transfer Studies (2007) (22)
HCO3− Secretion by Murine Nasal Submucosal Gland Serous Acinar Cells during Ca2+-stimulated Fluid Secretion (2008) (22)
Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice. (2017) (21)
Combination Adenovirus and Protein Vaccines Prevent Infection or Reduce Viral Burden after Heterologous Clade C Simian-Human Immunodeficiency Virus Mucosal Challenge (2017) (21)
Percutaneous Transendocardial Delivery of Self-complementary Adeno-associated Virus 6 Achieves Global Cardiac Gene Transfer in Canines. (2008) (21)
CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice. (2021) (20)
Pseudotyped adeno‐associated viral vector tropism and transduction efficiencies in murine wound healing (2012) (20)
Novel adenoviral vectors coding for GFP-tagged wtCFTR and ΔF508-CFTR: characterization of expression and electrophysiological properties in A549 cells (2004) (20)
Correction of Ureagenesis after Gene Transfer in an Animal Model and after Liver Transplantation in Humans with Ornithine Transcarbamylase Deficiency (1999) (20)
AAV8 capsid variable regions at the two-fold symmetry axis contribute to high liver transduction by mediating nuclear entry and capsid uncoating. (2014) (18)
Pseudotyped AAV Vector-Mediated Gene Transfer in a Human Fetal Trachea Xenograft Model: Implications for In Utero Gene Therapy for Cystic Fibrosis (2012) (18)
The ethical challenges of in utero gene therapy (2000) (18)
Activation of CFTR-specific T Cells in cystic fibrosis mice following gene transfer. (2007) (18)
AAV9 Provides Global Cardiac Gene Transfer Superior to AAV1, AAV6, AAV7, and AAV8 in the Mouse and Rat (2008) (18)
Gene therapy for dyslipidemia: a review of gene replacement and gene inhibition strategies (2010) (17)
Prediction of cellular immune responses against CFTR in patients with cystic fibrosis after gene therapy. (2007) (17)
Pharmacologically regulated regeneration of functional human pancreatic islets. (2005) (17)
AAV8 Gene Therapy Rescues the Newborn Phenotype of a Mouse Model of Crigler-Najjar. (2018) (17)
Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice. (2016) (17)
Influence of serotype, cell type, tissue composition, and time after inoculation on gene expression in recombinant adeno-associated viral vector-transduced equine joint tissues. (2012) (16)
The role of apoptosis in immune hyporesponsiveness following AAV8 liver gene transfer. (2013) (16)
Effects of Self-Complementarity, Codon Optimization, Transgene, and Dose on Liver Transduction with AAV8. (2016) (16)
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. (2013) (15)
Construction of gene transfer vectors based on simian adenovirus 7. (2011) (15)
It's time for gene therapy to get disruptive! (2012) (15)
A Single Injection of an Optimized Adeno-Associated Viral Vector into Cerebrospinal Fluid Corrects Neurological Disease in a Murine Model of GM1 Gangliosidosis (2020) (14)
Adeno-Associated Virus Serotype 9-Expressed ZMapp in Mice Confers Protection Against Systemic and Airway-Acquired Ebola Virus Infection. (2016) (14)
Standardized Method for Intra-Cisterna Magna Delivery Under Fluoroscopic Guidance in Nonhuman Primates. (2018) (14)
Optimized Adeno-Associated Viral-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques. (2018) (14)
Stable liver-specific expression of human IDOL in humanized mice raises plasma cholesterol. (2016) (14)
Recombinant adeno-associated virus preferentially transduces human, compared to mouse, synovium: implications for arthritis therapy (2004) (13)
A welcome animal model (1992) (13)
SARS vaccine: progress and challenge. (2005) (13)
Perspectives on best practices for gene therapy programs. (2015) (13)
Adeno-associated viral gene therapy corrects a mouse model of argininosuccinic aciduria. (2018) (12)
Wnt10b and Dkk-1 gene therapy differentially influenced trabecular bone architecture, soft tissue integrity, and osteophytosis in a skeletally mature rat model of osteoarthritis (2017) (12)
Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases (2013) (12)
Immune Responses in 101HEMB01, a Phase 1/2 Open-Label, Single Ascending Dose-Finding Trial of DTX101 (AAVrh10FIX) in Patients with Severe Hemophilia B (2017) (12)
α-Fetoprotein gene delivery to the nasal epithelium of nonhuman primates by human parainfluenza viral vectors. (2010) (11)
31st Annual Meeting and Associated Programs of the Society for Immunotherapy of Cancer (SITC 2016): part two (2016) (11)
Cystic fibrosis: strategies for gene therapy (1994) (11)
Adeno‐associated virus serotype 1‐based gene therapy for FTD caused by GRN mutations (2020) (11)
More from the modellers (1992) (10)
In vivo evaluation of adeno-associated virus gene transfer in airways of mice with acute or chronic respiratory infection. (2014) (10)
Media Reporting of the Emergence of the 1968 Influenza Pandemic in Hong Kong: Implications for Modern-day Situational Awareness (2009) (10)
Immunogenicity of an AAV-based, room-temperature stable, single dose COVID-19 vaccine in mouse and non-human primates (2021) (10)
Impact of intravenous infusion time on AAV8 vector pharmacokinetics, safety, and liver transduction in cynomolgus macaques (2016) (10)
Targeted retroviral gene transfer into the rat biliary tract (1996) (10)
Correction: AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina (2013) (10)
A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease. (2019) (10)
Adverse events in gene transfer trials and an agenda for the new year. (2008) (10)
Intrathecal Viral Vector Delivery of Trastuzumab Prevents or Inhibits Tumor Growth of Human HER2-Positive Xenografts in Mice. (2018) (10)
AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver (2018) (9)
Self-reactive CFTR T cells in humans: implications for gene therapy. (2013) (9)
Identification of the murine AAVrh32.33 capsid‐specific CD8+ T cell epitopes (2009) (8)
Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia (2021) (8)
Good news on the clinical gene transfer front. (2008) (8)
Effect of genetic transduction on in vitro canine endothelial cell prostanoid production and growth (1990) (8)
b-Defensin 1 Plays a Role in Acute Mucosal Defense against Candida albicans (2015) (8)
Prospects for gene therapy in ornithine carbamoyltransferase deficiency and other urea cycle disorders (1995) (8)
Genetic diseases, immunology, viruses, and gene therapy. (2014) (8)
Rescue of chimeric adenoviral vectors to expand the serotype repertoire (2007) (8)
Efficient Mitochondrial Import of Newly Synthesized Ornithine Transcarbamylase (OTC) and Correction of Secondary Metabolic Alterations in spfash Mice following Gene Therapy of OTC Deficiency (1999) (8)
Context-Specific Function of the Engineered Peptide Domain of PHP.B (2021) (8)
Efficacy and Safety of a Krabbe Disease Gene Therapy (2022) (7)
Increasing the specificity of AAV-based gene editing through self-targeting and short promoter strategies. (2020) (7)
Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Crigler-Najjar Syndrome (2018) (7)
Adeno-associated virus-mediated expression of human butyrylcholinesterase to treat organophosphate poisoning (2019) (6)
Challenges in the gene therapy commercial ecosystem (2017) (6)
Accurate and Rapid Sequence Analysis of Adeno-Associated Virus Plasmids by Illumina Next-Generation Sequencing. (2018) (6)
Autoimmunity, recessive diseases, and gene replacement therapy. (2010) (6)
Multiple recombinant adeno-associated viral vector serotypes display persistent in vivo gene expression in vector-transduced rat stifle joints. (2013) (6)
Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression (2022) (6)
5 Adenovirus Vectors (1999) (6)
Bulls, bubbles, and biotech. (2013) (6)
A pharmacologenic rheostat for gene therapy (1996) (6)
Representing the Limits of Judgment: Yvor Winters, Emily Dickinson, and Religious Experience (2007) (5)
Flushing out antibodies to make AAV gene therapy available to more patients. (2013) (5)
Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse model. (2021) (5)
Modified Adenovirus Prime-Protein Boost Clade C HIV Vaccine Strategy Results in Reduced Viral DNA in Blood and Tissues Following Tier 2 SHIV Challenge (2021) (5)
Adeno-Associated Virus Vector-Mediated Expression of Antirespiratory Syncytial Virus Antibody Prevents Infection in Mouse Airways. (2021) (5)
A call to arms for improved vector analytics! (2015) (5)
Human adenine phosphoribosyltransferase: Characterization from subjects with a deficiency of enzyme activity (1983) (4)
Functional CFTR Expression in Cystic Fibrosis Airway Epithelial Cells by AAV6.2-mediated Segmental Trans-splicing (2009) (4)
Motor Neuron Transduction After Intracisternal Delivery of AAV9 in a Cynomolgus Macaque (2015) (4)
Silurian-Niagaran Reef Belt Around the Michigan Basin -- A Synergistic Review (1982) (4)
Urolithiasis in a Large Kindred Deficient in Adenine Phosphoribosyltransferase (Aprt) (1985) (4)
A call to arms for improved vector analytics (2015) (4)
Abnormal polyamine metabolism is unique to the neuropathic forms of MPS: potential for biomarker development and insight into pathogenesis (2017) (4)
2017 Is the Year We Have Been Waiting For. (2017) (4)
Advancing translational research through the NHLBI Gene Therapy Resource Program (GTRP). (2013) (4)
Loss of transgene expression limits liver gene therapy in primates (2022) (4)
The first journal on human gene therapy celebrates its 25th anniversary. (2014) (4)
Regulation of gene therapy in china. (2006) (3)
Susceptibility to SIV Infection After Adenoviral Vaccination in a Low Dose Rhesus Macaque Challenge Model (2019) (3)
558. Anti-SARS Humoral and Cellular Immunity Evoked by an Adenovirus Vector Expressing Spike Glycoprotein from SARS Coronavirus (2004) (3)
Selection of rare event cells expressing β-galactosidase (1991) (3)
1048. Long-Term Regulated Gene Expression in Non-Human Primates Using a Single Non-Recombinogenic AAV Vector (2004) (3)
Isolating Human Monoclonal Antibodies Against Adeno-Associated Virus From Donors With Pre-existing Immunity (2020) (3)
Moving to the clinic with gene therapy through our new journal expansion, human gene therapy clinical development. (2012) (3)
Detection of reporter gene expression in murine airways. (2007) (3)
Progress in the commercial-scale production of adeno-associated viral vectors. (2009) (3)
Cone And Rod Transduction With Alternative AAV Serotypes In The Macula Of Non-human Primates (2011) (3)
The Vision of the Soul (2017) (3)
The History and Promise of Gene Therapy (2011) (3)
Liver-Directed Gene Therapy In the Treatment of Familial Hypercholesterolemia (1991) (3)
Safety of the Subretinal Delivery of RGX-314 AAV8-anti-VEGF Fab Gene Therapy in NHP: Retinal Structure Over One Year (2018) (3)
Identification and functional characterization in vivo of a novel splice variant of LDLR in rhesus macaques. (2011) (3)
Crispr/Cas9-Mediated In Vivo Gene Targeting Corrects Haemostasis in Newborn and Adult FIX-KO Mice (2016) (3)
Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer. (2013) (3)
Erratum: Oral vaccination of mice with adenoviral vectors is not impaired by preexisting immunity to the vaccine carrier (Journal of Virology (2003) 77:20 (10780-10789) (2005) (2)
Interview with Jean Bennett, MD, PhD. (2018) (2)
Correction to: ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing (2020) (2)
404. Roles of Inverted Terminal Repeats (ITRs) and Capsid Proteins from Novel NHP AAVs in rAAV Mediated Gene Transfer (2005) (2)
Use of Microbeads for Cell Transplantation (1989) (2)
Louis MacNeice's Struggle with Aristotelian Ethics (2006) (2)
The common variant of cystic fibrosis transmembrane conductance regulator is recognized by hsp 7 O and degraded in a pre-Golgi nonlysosomal compartment ( recombinant adenoviruses / molecular chaperones / immunoelectron microscopy ) (2005) (2)
Construction of Recombinant Adenovirus Encoding Human β-Glucuronidase cDNA under the Influence of Human β-Glucuronidase Promotor (1995) (2)
116. The Proposed AAV Serotypes 10 and 11 Serologically Cross-React with AAV8 and AAV4 (2006) (2)
High activity of an affinity-matured ACE2 decoy against Omicron SARS-CoV-2 and pre-emergent coronaviruses (2022) (2)
Motor neuron transduction after intracisternal delivery of AAV9 in a cynomolgus macaque. (2015) (2)
24. Sustained Expression with Partial Correction of Neutrophil Defects 5 Years After Intramuscular rAAV1 Gene Therapy for Alpha-1 Antitrypsin Deficiency (2016) (2)
AAV-Mediated Retinal Transfer Of Anti-angiogenic Genes In A Murine Model Of Retinopathy Of Prematurity (ROP) (2002) (2)
The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute. (2017) (2)
There and Back Again: Mitchell Finer on the Journey of Biotech from Start-Up to Success. (2015) (2)
Poetic Jansenism: Religious and Political Representation in Denis Devlin's Poetry (2007) (2)
Ex vivo gene therapy directed to liver in a patient with familial hypercholesterolemia (1994) (2)
Ancient Beauty, Modern Verse: Romanticism and Classicism from Plato to T. S. Eliot and the New Formalism (2015) (1)
338. Therapeutic Levels for |[alpha]|1-Antitrypsin Following Intrapleural Administration of a Non-Human Primate Serotype rh10 AAV Vector Expressing |[alpha]|1-Antitrypsin (2004) (1)
Gene therapy for mucopolysaccharidosis VII: evaluation of intrathecal rAAV vectors in the canine model (2014) (1)
90. Identification of an Adeno-Associated Virus Binding Epitope for AVB Sepharose Affinity Resin (2015) (1)
The Formal and Moral Challenges of T. S. Eliot’s Murder in the Cathedral (2015) (1)
A Correction to the Research Article Titled: "Intranasal Antibody Gene Transfer in Mice and Ferrets Elicits Broad Protection Against (2013) (1)
Moving Forward after Two Deaths in a Gene Therapy Trial of Myotubular Myopathy (2020) (1)
Adeno_Associated Virus (AAV)-Mediated Transduction of the Macula in Non-Human Primates (NHPs): Cell-Specificity, Stability and Safety of Transduction After Subretinal Delivery of AAV2 and AAV2/8 Vectors (2007) (1)
The Birth of Human Gene Therapy Methods (2011) (1)
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques (2022) (1)
Constitutive and Regulated Expression in the Systemic Delivery of Erythropoietin Following Skeletal Muscle Transduction with DNA Viral Vectors (1999) (1)
The Realism of Helen Pinkerton (2009) (1)
John Paul II’s Letter to Artists and the Force of Beauty (2014) (1)
Adenoviruses as Vectors for Human Gene Therapy (2002) (1)
Strategies for Selection of AAV Vectors for Administration to Liver: Studies in Nonhuman Primates (2016) (1)
Receptor-mediated gene delivery and expression in hepatocytes (1990) (1)
Is This Gene Therapy's Breakout Moment? (2013) (1)
The LDL receptor is not required for normal catabolism of Lp(a) in humans (1994) (1)
Breakthrough to Bedside: Bringing Gene Therapy to Neuromuscular Diseases. (2019) (1)
The Encyclopedia of Epic Films (2014) (1)
Gene transfer to primate liver using second generation recombinant adenovirus (1995) (1)
141. Adenovirus Vaccine to Ebola Virus: Mechanism of Protection and Impact of Pre-Existing Immunity to the Vaccine Carrier (2005) (1)
Doctrinal Development and the Demons of History: The Historiography of John Henry Newman (2006) (1)
847. Adeno-Associated Virus Capsids from Divergent AAV Clades and Subgroup C Adenovirus Capsids Share a Common Microtubule-Binding Mechanism Relevant to Virus Trafficking (2005) (1)
611. Induction of Persistent Passive Immunity Against Anthrax Toxin by an Adeno-Associated Virus Type rh10 Vector Expressing Anti-Protective Antigen Antibody (2006) (1)
1081. Sustained Correction of Disease in Na|[iuml]|ve and AAV2-Pretreated Hemophilia B Dogs Using Pseudotyped AAV Vectors (2005) (1)
Alternative Start Sites Downstream of Non-Sense Mutations Drive Antigen Presentation and Tolerance Induction to C-Terminal Epitopes (2017) (1)
Cycling at the Frontiers of Gene Therapy. (2019) (1)
From Plymouth Rock to Palo Alto: The New England literary tradition and its American critics (2014) (1)
853. Persistent Expression of Single Chain Antibodies Mediated by AAV5 and AAVrh.10 Vectors (2005) (1)
406. Role of MyD88 Signaling Pathway in the Host Immune Responses in Adenoviral Vector-Mediated Gene Therapy (2005) (1)
Identification of Novel Adeno-Associated Virus Serotypes for Use as Vectors (2006) (1)
1015. Induction of Protective Immunity to Anthrax Lethal Toxin with a Chimpanzee Adenovirus-Based Vaccine Carrier in the Presence of Pre-Existing Anti-Human Adenovirus Immunity (2004) (1)
Novel and scalable approach to research grade AAV vector manufacturing and separation of distinct AAV serotypes (2009) (1)
Normal parameters of the full field ERG recorded with bipolar electrodes in Cynomolgus Macaque (Macaque fascicularis) (2017) (1)
Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase. (2021) (1)
206. Frequency and Analysis of Liver Tumors in AAV-Treated B6C3F1 Hybrid Mice (2006) (1)
Correction for Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy, by Mark L. Brantly, Jeffrey D. Chulay, Lili Wang (2009) (1)
60. Engineering AAV Vector for the Delivery of Human BuChE to Protect Against Exposure to Organophosphates (2016) (1)
Luigi Naldini on His Lifelong Involvement with the Development of Gene Therapy (2016) (1)
779. Genetic Delivery of an Anti-RSV Antibody Protects Against Pulmonary Infection with RSV in Mice (2006) (1)
Correction for Desplats et al., Inclusion formation and neuronal cell death through neuron-to-neuron transmission of α-synuclein (2009) (1)
161. Novel pseudotyped AAV vectors for hemophilia B gene therapy (2004) (1)
Isolation and characterization of adenoviruses persistently shed from the gastro- intestinal tract of non-human primates (2012) (1)
Safety of subretinal delivery of RGX-314 (AAV8-anti-VEGF Fab) in the non-human primate as assessed by full-field ERG (2018) (1)
BRIAN COFFEY, JACQUES MARITAIN AND THE RECOVERY OF THE ‘THING’ (2010) (1)
When Selecting an Adeno‐associated Viral Vector Serotype, Cell Monolayer Transduction Efficiency Does Not Accurately Predict Tissue Transduction Efficiency in Equine Synovial Tissues (2009) (1)
Anti-CD4 Antibody Permits Readministration of Recombinant Adenovirus to Correct Ornithine Transcarbamylase Deficiency (OTCD) in the Sparse Fur(spf/Y) Mice. • 638 (1997) (1)
Interview with Inder Verma, PhD. (2016) (1)
Adenosine Deaminase Deficiency with (1983) (1)
218. Nonhuman Primate Models for Retinal and Choroidal Neovascularization Using AAV2-Mediated Overexpression of Vascular Endothelial Growth Factor (2004) (1)
1089. Examine the Cross-Reactivity of Vector-Specific T Cells between Human Adenoviral H5 and Simian Adenoviral C7 Vector in Mice (2005) (1)
The Next Chapter (2015) (1)
Murine and Nonhuman Primate Lungs Responses to Adenovirus Vectors Delivered to Role of E4 in Eliciting CD4 T-Cell and B-Cell (2013) (1)
Evaluating the impact of systemic AAV9.cIDUA administration on brain pathology in MPS I dogs (2015) (1)
Stress echocardiography in children with homozygous familial hypercholesterolemia predicts severe coronary artery disease (1996) (1)
Modified AAVrh.64 capsids, compositions containing them and their uses (2006) (0)
Development of a novel gene therapy for Fabry disease: Engineered alpha-galactosidase A transgene for improved stability (2021) (0)
Clinical Evaluation of a Recombinant Adeno-Associated Virus (rAAV) Alpha-1 Antitrypsin (AAT) Gene Therapy Vector. (2009) (0)
Recombinant AAV for the manufacture of a medicament for gene therapy of muscle cells (1997) (0)
Assessment of metabolic abnormalities from the cerebellar region of the brain of a canine model of mucopolysaccharidosis type I ( MPS I ) using in vivo 1 H MRS (2013) (0)
Nucleic acid sequences from the adeno-associated virus serotype 1, as well as vectors and host cells containing these (1999) (0)
Refining the tools for gene therapy of hemophilia (2006) (0)
139. Priming and Boosting Efficiency of Chimp and Human Adenoviral Vectors in the Setting of Pre-Existing Immunity in Non-Human Primates (2005) (0)
569. Increased Antigen-Specific Humoral Response by Adenoviral Vector Prime and Adeno-Associated Viral Vector Boost (2004) (0)
Correction to: ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing (2020) (0)
Aav7 viral vectors for targeted delivery of rpe cells (2009) (0)
508. Impact of Sex on Serum |[alpha]|1-Antitrypsin Levels Following Intrapleural and Intravenous Administration of AAVrh.10 Vectors to Mice (2005) (0)
1016. Novel Chimeric Adenovirus Vaccine Vectors (2004) (0)
977. Differential Host T Cell Response to the Capsid of AAV2 and 8 in Muscle-Directed Gene Transfer in Murine and Non-Human Primate Models (2006) (0)
Analysis of the AAVrh. 10 Capsid Structure and Its Antigenic and Receptor Interactions (2017) (0)
Abstract 71: AAV-Mediated Expression of Human IDOL in Mouse Liver Increases LDL Cholesterol in a Dose-Dependent Fashion (2014) (0)
In memory of Sonia Skarlatos, PhD (1953-2013). (2013) (0)
Cell lines and constructs that are useful in the production of E1-deleted adenoviruses in the absence of replication competent adenovirus (2000) (0)
101. Molecular Mechanism Responsible for High Level Transgene Expression in Murine and Nonhuman Primate Liver with the Second Generation of Novel AAV Serotype Vectors (2006) (0)
Systemic IFN-b gene therapy results in long-term survival in mice with established colorectal liver metastases (2018) (0)
ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing (2020) (0)
Targeted release of genes encoding secretory proteins (1992) (0)
Dose preparation sheet (2016) (0)
The structure of the neurotropic AAVrh.8 viral vector (2016) (0)
Procédé évolutif de production d'aav (2007) (0)
346. AAV9 Delivery into Cerebrospinal Fluid Corrects CNS Disease in a Murine Model of Mucopolysaccharidosis Type II (2016) (0)
The technology of gene therapy. (2011) (0)
Adenovirus it comprising a hexónica capsid protein simian adenovirus SAdV-E 39 and uses thereof (2008) (0)
CHAPTER 53 – Gene Therapy (2009) (0)
Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases (2022) (0)
785. A Novel Adeno Associated Virus Vector That Efficiently Transduces Airway Epithelium In Vivo and Can Be Re-Administered (2006) (0)
684. Improved Performance of AAV6 in Airways In Vivo (2006) (0)
Novel Approach to Intermediate Scale AAV Vector Manufacturing and Separation of Distinct AAV Entities on Iodixanol Gradients (2009) (0)
692. A novel AAV vector for the treatment of cystic fibrosis airway disease (2004) (0)
Immunogenicity of a pre-erythrocytic-stage malaria insert ME-trap encoded by novel simian adenoviral vectors (2005) (0)
Candida albicans Defense against -Defensin 1 Plays a Role in Acute Mucosal β (2015) (0)
Transgenic ablation system mediated inducible adeno-associated virus (2012) (0)
Rapamycin-inducible Secretion of a Therapeutic Protein in the Eye After Transduction With Viral Vectors (2002) (0)
607. Novel Adeno-Associated Viruses as Vaccine Carriers for HIV-1: Evaluation in Non-Human Primates (2006) (0)
Breast milk lactoferrin regulates intestinal gene expression by binding bacterial DNA but not genomic DNA (2003) (0)
Clades and sequences of adeno-associated virus (AAV) vectors containing them, and uses thereof (2002) (0)
Large Animal Models of Lysosomal Storage Diseases: Lessons on the Limits of Gene/Enzyme Therapy (2012) (0)
978. Reduced Immune Response to Adenovirus Vector Formulated with the Anti-Inflammatory Liposome DS/DOPE (2006) (0)
77. TLR and Myd88 Signaling Pathway Is Required for the Full Induction of Host Adaptive Immune Responses to Adenoviral Vectors and Transgenes (2006) (0)
Clades of adeno-associated virus (AAV) sequences, vectors containing them, and uses thereof (2004) (0)
Correction of hyperglycemia by adenoviral vector-mediated insulin gene expression in the murine pancreas (2000) (0)
696. TLR9 Signaling Mediates Transgene Antibody Formation (2016) (0)
Adeno-associated virus (aav) clade f vector and uses therefor (2018) (0)
GENE THERAPY IN THE ORNITHINE TRANSCARBAMYLASE DEFICIENT MOUSE PREVENTS CLINICAL SEQUELAE OF NITROGEN CHALLENGE. ▴ 886 (1996) (0)
Compositions and methods for the adjustment free production of recombinant adenoasociated viruses. (2000) (0)
Phase I trial of adenoviral gene transfer in ornithine transcarbamylase deficiency † 731 (1998) (0)
In Vivo Gene Therapy Corrects Neurochemical Abnormalities in the Ornithine Transcarbamylase (OTC) Deficient Sparse Fur (spf/Y) Mouse. ♦ 1748 (1997) (0)
The oxidation of chalcone catalysed by peroxidase : a thesis presented in partial fulfilment of the requirements for the degree of Doctor of Philosophy in Plant Science at Massey University (1971) (0)
Use of recombinant adeno-associated virus in the preparation of a medicament for gene therapy by muscle cells. (1997) (0)
In Utero AAV Vector-Mediated Gene Delivery To The Retina (2002) (0)
Measuring Ureagenesis In Vivo with Stable Isotopes Provides a Tool to Assess Efficacy of Gene Therapy for Ornithine Transcarbamylase Deficiency ♦ 727 (1998) (0)
Adeno-Associated Virus(AAV)-Mediated Expression of Ornithine Transcarbamylase (OTC) in OTC Deficient spf-ash mMice (1999) (0)
604. Comparative Analysis of Different Methods for Quantitative Evaluation of Neutralizing Antibody to Ebola Virus in a Biosafety Level 2 or 4 Environment (2006) (0)
GENE THERAPY FOR TREATING WILSON'S DISEASE (2018) (0)
10308 Mutant Forms of Hypoxanthine-Guanine Phosphoribosyltransferase (2001) (0)
Studies on the phenolic compounds of apple leaf tissue (Malus pumila Mill.) : a thesis presented in partial fulfilment of the requirements for the degree of Master of Horticultural Science in Plant Science (1967) (0)
525. CD8+ T Cell Tolerance To Epitopes Downstream of Non-Sense Mutations (2015) (0)
Comparative toxicity of E4 deleted vectors expressing human ornithine transcarbamylase (OTC) cDNA ♦ 730 (1998) (0)
Formation of Newly Synthesized Adeno-Associated Virus Capsids in the Cell Nucleus (2014) (0)
A Journey in the Development of Gene Therapy for Inherited Disorders of the Bone Marrow (2015) (0)
A MOLECULAR SURVEY OF HPRT DEFICIENCY: 48 (1985) (0)
From Being to Faith: The Poems of Helen Pinkerton (2012) (0)
Prospects of a Catholic Literary Revival (2017) (0)
University Flunk-Out to Genomics Pioneer: An Interview with George Church, PhD. (2018) (0)
Carl June Speaks of His Pioneering Efforts That Led to the First Food and Drug Administration-Approved Gene Therapy Product. (2017) (0)
Supporting US public health experts (2020) (0)
"I Bought and Praised but Did Not Read Aquinas": T.S. Eliot, Jacques Maritain, and the Ontology of the Sign (2010) (0)
A structural analysis of myths from the north-east frontier of India. (1970) (0)
Human Gene Therapy Clinical Development: Where the Academy and Industry Meet. (2015) (0)
The Worcester Liber Albus: Glimpses of Life in a Great Benedictine Monastery in the Fourteenth Century (2017) (0)
695. An AAV8 Mutant with Better Transduction in Murine Muscle and Nasal Airway Than AAV8 (2016) (0)
Tachi Yamada: An Academic, Drug Developer and Humanist. (2018) (0)
A new editorial team. (2011) (0)
Catholic Modernism and the Irish "Avant-Garde" (2006) (0)
Socrates in Hell: Anthony Hecht, Humanism, and the Holocaust (2011) (0)
A Birds-Eye View: An Interview with Nick Leschly. (2019) (0)
Thomas MacGreevy Reads T.S. Eliot and Jack B. Yeats: Making Modernism Catholic (2006) (0)
A New Open Access Partner (2012) (0)
The Story of RNA Interference as a New Therapeutic Paradigm from Nobel Laureate Craig Mello. (2017) (0)
STYLE AND SUBSTANCE: T. S. ELIOT, JACQUES MARITAIN, AND NEO-THOMISM (2010) (0)
Author Correction: Supporting US public health experts (2020) (0)
Book Review: Aesthetic Perception: A Thomistic PerspectiveAesthetic Perception: A Thomistic Perspective. By O'ReillyKevin E., Dublin: Four Courts Press, 2007. ISBN 978-1-84682-027-4. Pp. 131. $55.00. (2010) (0)
Gene Therapy Entering the Land of Oz. (2018) (0)
The Past, Present, and Future of Gene Therapy from Nobel Laureate David Baltimore. (2017) (0)
Jurassic Park, Gene Therapy, and Neuroscience: An Interview with Feng Zhang, PhD. (2017) (0)
Jurassic Park, Gene Therapy, and Neuroscience (2017) (0)
Luigi Naldini on His Lifelong Involvement with the Development of Gene Therapy. (2016) (0)
Formal Protests (2014) (0)
The Vision of the Soul: Truth, Beauty, and Goodness in the Western Tradition (2017) (0)
Societies, Makeovers, and Awards (2009) (0)
Recent Developments with the Journal (2004) (0)
The RAC Retires after a Job Well Done (2019) (0)
The Catholicity of Beauty (2017) (0)
Interview with Katherine A. High, MD. (2016) (0)
506. Transductional and Genome Biodistribution of Novel AAV Serotype Vectors after Systemic Delivery and Their Liver Specific Dose Response Profiles in Mice (2005) (0)
Lignees de cellules et produits d'assemblage servant a l'obtention d'adenovirus a deletion e-1 en l'absence d'adenovirus a capacite de replication (2000) (0)
890. Improvement of Behavior and Mortality Following CNS Administration of AAVrh.10hCLN2 to CLN2 −/− Mice, a Model LINCL (Battan Disease) a Fatal Childhood Neurological Disorder (Batten Disease) (2006) (0)
484. Murine Liver as a Model for AAV Replication, Recombination and Evolution (2006) (0)
Antibody Mice Treated with a Nondepleting CD 4 Vectors in Lungs of Human CD 4 Transgenic Repeated Administration of Adenoviral (1999) (0)
Gene delivery to the heart and liver using AAV serotype 9 : implications for cardiovascular disease and hemophilia (2005) (0)
Infection in Mouse Lung influenzae Haemophilus Immune Responses to Toll-Like Receptor 4 Mediates Innate (2001) (0)
Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors (2022) (0)
Recollections from a Pioneer Who Provided the Foundation for the Success of Gene Therapy in Treating Severe Combined Immune Deficiencies. (2016) (0)
660. Quantitative Comparison of AAV Serotypes AAV2, AAV5, and AAVrh.10 Efficiency for CNS Gene Therapy Following Intracranial Gene Delivery (2005) (0)
in Human Cancer Cells β Signaling Pathway by Interferon Apoptosis-inducing Ligand and the Cell-Surface Death Stat 1-dependent Induction of Tumor Necrosis Factor-related Updated (2003) (0)
812. Non-Human Primate Serotype rh.10 AAV Directed Therapeutic Serum Levels of |[alpha]|1-antitrypsin Following Intrapleural Administration in Mice with Pre-Existing Immunity to AAV (2005) (0)
Preparation of Nonhuman Primate Eyes for Histological Evaluation After Retinal Gene Transfer. (2018) (0)
Neonatal Mice Product-Specific Antibody Responses in Vaccine Carriers Induce Transgene Mucosally Delivered E1-Deleted Adenoviral (2003) (0)
Sequences adeno-associated virus serotype 8 (AAV) vectors containing them and uses thereof (2002) (0)
568. Comparison of Antigen-Specific Immune Responses Elicited by Recombinant Simian Adenoviral Vectors with Deletions in Either E1, or E1/E3, or E1/E4 Regions (2004) (0)
744. Novel Vectors for Gene Therapy: Covering the Spectrum of Primate AAV Diversity (2006) (0)
Adeno-associated virus vector-mediated gene therapy can effectively treat CNS and cardiac lesions and induce immune tolerance to the therapeutic enzyme in large animal models of mucopolysaccharidosis type I (2015) (0)
Compositions and methods for the helper production of recombinant adeno-associated virus (2000) (0)
Diversity of adenoviruses in humans and in non-human primates (2012) (0)
The RAC Retires After a Job Well Done. (2018) (0)
Secretion by Murine Nasal Submucosal Gland Serous Acinar Cells during Ca (2008) (0)
732. A Novel Double-Pseudotyped Viral Vector for Gene Therapy (2004) (0)
In the Beginning: Reflections on the Genesis of Molecular Therapy. (2010) (0)
112. Strong Transgene-Specific Immune Responses Can Be Elicited with Novel Adeno-Associated Virus Vectors in Mice (2005) (0)
TBG . mLDLR and AAV 8 . TBG . hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia (2017) (0)
189. Therapeutic Gene Transfer as a Treatment Option for Age-Related Macular Degeneration (2016) (0)
Determining the Minimally Effective Dose of a Clinical Candidate Adeno-Associated Virus Vector in a Mouse Model of Hemophilia A (2021) (0)
872. Chimpanzee Derived Adenoviral Vectors Transduce and Activate Human Dendritic Cells (2005) (0)
900. Specific AAV Serotypes Stably Transduce Hippocampal and Cortical Cultures with High Efficiency and Low Toxicity (2006) (0)
Groups of adeno-associated virus (AAV), SEQUENCES, BODIES OF AND USES containing these (2017) (0)
INFECTIOUS DISEASES & VACCINES (2006) (0)
Evaluation of a novel delivery strategy for pulmonary gene transfer in a fetal mouse model using AAV6.2 (2009) (0)
A method for the identification of unknown sequences adeno-associated virus (AAV) and a kit for the method. (2002) (0)
82. Mapping the Humoral Immune Response to AAV by Molecular Docking and Cryo-Electron Microscopy for the Design of Next-Generation AAV Vectors (2016) (0)
Corrigendum to "Biology of AAV Serotype Vectors in Liver-Directed Gene Transfer to Nonhuman Primates". (2006) (0)
Adenovirus comprising a simian adenovirus E-SAdV-39-capsidhexonprotein and uses thereof (2008) (0)
146. T Cell Response Against the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene (2005) (0)
646. Isolation and Evaluation of Novel Anti-AAV2 and AAV3B Antibody Clones from a Human Donor (2015) (0)
Formation of newly synthesized adeno-associated virus capsids in the cell nucleus. (2014) (0)
1101. Rapid and Long-Term Protection Against Anthrax Lethal Toxin (2006) (0)
Glybera's Story Mirrors that of Gene Therapy (2013) (0)
227. A Dose-Escalating Preclinical Study to Determine the Efficacy, MED, and Safety of a Clinical Candidate Vector in a Mouse Model of Hemophilia B (2016) (0)
Adeno-Assocated Virus ( AAV ) Serotype 9 Provides Global Cardiac Gene Transfer Superior to AAV 1 , AAV 6 , AAV 7 , and AAV 8 in the Mouse and Rat (2018) (0)
368. Pseudotyping HIV Vector with the Spike Envelope Protein of SARS-CoV for Studying Viral Tropism, Immunology and Gene Therapy Applications (2004) (0)
373. Simian Adenoviral Vector Based-Vaccine Fully Protect Against Ebola Virus Even in the Presence of Pre-Existing Immunity to Human Adenovirus (2004) (0)
Nucleic acid sequences of the adeno-associated virus serotype i, and vectors and host cells containing these (1999) (0)
Methods: clinical trial design and outcome measures (2016) (0)
AAV Vectors for Infectious Diseases (2013) (0)
Method for direct extraction and amplification of integrated cellular DNA virus tissue. (2003) (0)
Results of mechanistic substudy (2016) (0)
Comparative analysis of multiple AAV vector serotypes in the mouse brain (2007) (0)
In vivo gene therapy of the equine distal extremity with recombinant adeno-associated viral vectors for the treatment of laminitis (2013) (0)
9. AAV-Mediated Gene Transfer as a Therapeutic Approach for Familial LCAT Deficiency (2015) (0)
RH10 serotype of adeno-associated virus (AAV) (2002) (0)
Safety and adverse events (2016) (0)
111. AAV7, 8, and 9 Are More Efficient and Less Immunogenic Vectors for Muscle-Directed Gene Therapy for Hemophilia B (2005) (0)
Method for increasing the functionality of an AAV vector (2006) (0)
AAVrh.64 modified capsids, compositions containing them and uses thereof (2006) (0)
, Receptor 5 Recognition of Flagellin by Toll-Like Infection via Pseudomonas aeruginosa Human Airway Epithelial Cells Sense (2005) (0)
Human fetal trachea xenografts recapitulate normal development: implications for fetal gene therapy using pseudotyped AAV vectors (2003) (0)
Liver-Directed Gene Therapy for Mucopolysaccharidosis Type I (MPS I) (2012) (0)
Intrathecal injection of lentiviral vector results in high expression in the brain of mucopolysaccharidosis VII dogs but the pattern of expression is different than for AAV9 or AAV-rh10 (2015) (0)
90. Evaluation of Novel Gene Transfer Vectors Derived from Infectious Molecular Clones of Primate AAVs (2004) (0)
Comparison of different rAAV serotypes for transduction of dopaminergic neurons in rat substantia nigra (2009) (0)
Phase 2 Clinical Trial Of A Recombinant Adeno-Associated Virus (RAAV) Alpha-1 Antitrypsin (AAT) Gene Therapy Vector (2011) (0)
Sequential supply of immunogenic molecules by administrations of an adenovirus and an adeno-associated virus. (2005) (0)
A method for identifying adeno-associated virus (AAV) sequences and kit for carrying out the method (2002) (0)
United States Patent ( 19 ) Wilson et al . ( 54 ) NON-HUMAN ANIMAL MODEL OFA HUMAN AIRWAY (2017) (0)
Patient information sheets (2016) (0)
1092. Adenovirus-Based Vaccine Prevents Pneumonia in Ferrets Challenged with the SARS Coronavirus (2005) (0)
Adeno associated virus. (1995) (0)
Subretinal delivery of RGX-314 AAV8-anti-VEGF Fab gene therapy in NHP (2017) (0)
rAAV9 airway delivery results in effective knockdown of mutant alpha 1-antitrypsin in the liver while upregulating wildtype alpha 1-antitrypsin in the lung (2012) (0)
699. Effective AAV9 Vector Delivery to Nasal Mucosa for Protection Against Airborne Challenge with Influenza A and B (2016) (0)
33* Optical imaging of calcium-evoked fluid secretion by murine airway submucosal gland serous acinar cells (2007) (0)
490. Long Term Expression and Biodistribution of HIV Vector Pseudotyped with Deletion Mutant of the Ebola Envelope Glycoprotein Following Instillation in Airway of Nonhuman Primates (2004) (0)
976. TLR and MyD88 Signaling Mediates Innate Immune Responses to Adenoviral Vectors in Mouse (2006) (0)
Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase (2023) (0)
Method for the direct recovery and amplification of integrated viral DNA from cellular tissue (2003) (0)
Results of clinical efficacy outcomes (2016) (0)
Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors (2022) (0)
Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them and their uses (2002) (0)
Preclinical development of a platform for enzyme therapy in the CNS of MPS I and MPS II patients based on intrathecal AAV delivery (2018) (0)
500. Biology of Novel AAV Serotype Vectors in the Liver Directed Gene Transfer to Nonhuman Primates (2005) (0)
503. Mutational Strategy To Enhance or Rescue Functionality of Existing AAV Vectors (2005) (0)
The Garden (2020) (0)
Human Gene Therapy: A New Beginning (1998) (0)
Cystic Fibrosis Questionnaire – Revised quality-of-life questionnaire (2016) (0)
Novel Adeno-Associated Virus Serotypes Efﬁciently Transduce Murine Photoreceptors (cid:1) (2007) (0)
595. Nasal Delivery of Adenovirus Expressing the Ebola Glycoprotein Protects Mice Against Ebola Virus in the Presence of Preexisting Immunity to the Vaccine Carrier (2006) (0)
760. Optimized AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice and Cynomolgus Macaques (2016) (0)
Simian adenoviruses subfamily B SADV-28, -27, -29, -32, -33 and -35 and uses thereof (2008) (0)
609. Acute-Phase Toxicity Following Systemic Administration of Non-Human Primate Derived Adenoviral Vectors to Mice (2006) (0)
481. CRISPR/Cas9-Mediated In Vivo Genome Editing to Correct the OTC spfash Mutation in Newborn Mice (2016) (0)
Method scalable AAV production (2007) (0)
Carriers Heterologous E 1-Deleted Adenoviral Vaccine withAntigen through a Prime Boost Regimen T Cells to an HIV-1 + Induction of CD 8 (2003) (0)
Method for purifying viral vectors have proteins that bind to sialic acid (2002) (0)
Neonatal Fc receptor inhibition enables adeno-associated virus gene therapy despite pre-existing humoral immunity. (2023) (0)
Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model (2023) (0)
Urinary glycosaminoglycan concentration as a biomarker for effectiveness of enzyme replacement and gene therapy in large animal models of mucopolysaccharidoses (2016) (0)
Adenovirus-AAV hybrid virus and method of use thereof. (1995) (0)
88. AAV Clades: Their Ability To Recombine and Cross Species-Barriers (2004) (0)
498. Targeted Gene Transfer to Lung Airway Epithelium Using Plasmid or Adenoviral Vectors Formulated with an Anti-Inflammatory Dexamethasone-Spermine Conjugate (2004) (0)
Characterizing Complex Populations of Endogenous Adeno-associated Viruses by Single-Genome Amplification. (2022) (0)
Impact of Single Amino Acid Modifications of Adeno-Associated Virus (AAV) Capsid and Viral Dose on Transduction Patterns After Transuterine Subretinal Delivery of AAV Vectors to the Fetal Retina (2009) (0)
A Journey in the Development of Gene Therapy for Inherited Disorders of the Bone Marrow. (2015) (0)
174. Liver Fibrosis in Aged OTC-KO Heterozygotes and Successful Correction by AAV8-Mediated Gene Therapy (2015) (0)
Repeated administration of adenoviral vectors in lungs of human CD4 transgenic mice treated with blocking CD4 antibody (1999) (0)
Trans-Splicing AAV Vector System Expands the Packaging Capacity Of AAV Gene Therapy Vectors for Delivery of Large Transgenes to the Retina (2002) (0)

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