Terence R. Flotte

Terence R. Flotte's AcademicInfluence.com Rankings

Terence R. Flotte

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#12524

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#6033

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#7510

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Philosophy

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Terence R. Flotte'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

Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. (2008) (942)
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics (2008) (687)
Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. (1993) (494)
Defective regulation of outwardly rectifying Cl− channels by protein kinase A corrected by insertion of CFTR (1992) (417)
Mutational Analysis of the Adeno-Associated Virus Type 2 (AAV2) Capsid Gene and Construction of AAV2 Vectors with Altered Tropism (2000) (383)
Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. (1993) (349)
Adeno-associated virus vectors for gene therapy. (1995) (331)
Clinical gene therapy using recombinant adeno-associated virus vectors (2008) (320)
Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results. (2011) (305)
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy (2009) (303)
A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. (1996) (255)
A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. (2002) (252)
α-1 Antitrypsin Inhibits Caspase-3 Activity, Preventing Lung Endothelial Cell Apoptosis (2006) (249)
Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study. (2003) (241)
Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. (1994) (219)
Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus (1998) (216)
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. (2006) (215)
A novel antiapoptotic role for alpha1-antitrypsin in the prevention of pulmonary emphysema. (2006) (212)
Safety and Biological Efficacy of an Adeno‐Associated Virus Vector–Cystic Fibrosis Transmembrane Regulator (AAV‐CFTR) in the Cystic Fibrosis Maxillary Sinus (1999) (212)
Long-term, efficient inhibition of microRNA function in mice using rAAV vectors (2012) (198)
The Short Apical Membrane Half-life of Rescued ΔF508-Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Results from Accelerated Endocytosis of ΔF508-CFTR in Polarized Human Airway Epithelial Cells* (2005) (195)
Controlling neuropathic pain by adeno-associated virus driven production of the anti-inflammatory cytokine, interleukin-10 (2005) (181)
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. (2006) (180)
Improved method of recombinant AAV2 delivery for systemic targeted gene therapy. (2002) (173)
Latent Adeno-Associated Virus Infection Elicits Humoral but Not Cell-Mediated Immune Responses in a Nonhuman Primate Model (1999) (170)
Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy. (2016) (167)
Gene expression from adeno-associated virus vectors in airway epithelial cells. (1992) (165)
Repeated intrathecal injections of plasmid DNA encoding interleukin-10 produce prolonged reversal of neuropathic pain (2006) (159)
Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. (2004) (156)
Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here? (2019) (156)
Gene Therapy Progress and Prospects: Recombinant adeno-associated virus (rAAV) vectors (2004) (151)
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. (2006) (151)
alpha-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosis. (2006) (149)
MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression (2010) (146)
IL-10 suppresses chemokines, inflammation, and fibrosis in a model of chronic renal disease. (2005) (145)
Adeno-associated virus vector-mediated IL-10 gene delivery prevents type 1 diabetes in NOD mice (2001) (142)
Systemic Overexpression of IL-10 Induces CD4+CD25+ Cell Populations In Vivo and Ameliorates Type 1 Diabetes in Nonobese Diabetic Mice in a Dose-Dependent Fashion 1 (2003) (139)
CIC-2: a developmentally dependent chloride channel expressed in the fetal lung and downregulated after birth. (1995) (138)
Both CFTR and outwardly rectifying chloride channels contribute to cAMP-stimulated whole cell chloride currents. (1994) (130)
Gene therapy: The first two decades and the current state‐of‐the‐art (2007) (128)
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. (2013) (127)
Lack of cystic fibrosis transmembrane conductance regulator in CD3+ lymphocytes leads to aberrant cytokine secretion and hyperinflammatory adaptive immune responses. (2011) (122)
Effect Of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle (2001) (119)
Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver (2003) (118)
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. (2010) (113)
An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction. (1995) (108)
Repeated Delivery of Adeno-Associated Virus Vectors to the Rabbit Airway (1999) (107)
Moving forward after two deaths in a gene therapy trial of myotubular myopathy. (2020) (107)
DNA-dependent PK inhibits adeno-associated virus DNA integration (2004) (104)
Interleukin 10 attenuates neointimal proliferation and inflammation in aortic allografts by a heme oxygenase-dependent pathway. (2005) (103)
Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles (2012) (102)
Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice (2020) (96)
Adeno-associated virus-mediated IL-10 gene therapy inhibits diabetes recurrence in syngeneic islet cell transplantation of NOD mice. (2003) (93)
Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. (2005) (91)
Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients (2000) (89)
IL-10 regulation of lupus in the NZM2410 murine model (2006) (88)
A Phase I/II Study of tgAAV-CF for the Treatment of Chronic Sinusitis in Patients with Cystic Fibrosis. Stanford University, Stanford, California (1998) (87)
Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin (rAAV-SERPINA1) vectors in a nonhuman primate model: safety and immunologic aspects. (2002) (87)
It Is Time for Zero Tolerance for Sexual Harassment in Academic Medicine. (2017) (84)
Virus-Based Gene Delivery Systems (2002) (83)
Immune responses to recombinant adeno-associated virus vectors: putting preclinical findings into perspective. (2004) (83)
Deposition and expression of aerosolized rAAV vectors in the lungs of Rhesus macaques. (2002) (80)
Use of the NADH-Quinone Oxidoreductase (NDI1) Gene ofSaccharomyces cerevisiae as a Possible Cure for Complex I Defects in Human Cells* (2000) (79)
In Vivo Complementation of Complex I by the Yeast Ndi1 Enzyme (2006) (79)
Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neurons. (2008) (76)
A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice. (2013) (76)
Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno‐associated virus (rAAV1) vector (2006) (71)
Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma? (2015) (70)
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency (2017) (70)
Gene therapy in cystic fibrosis. (1999) (70)
Histone Deacetylase Inhibitor (HDACi) Suberoylanilide Hydroxamic Acid (SAHA)-mediated Correction of α1-Antitrypsin Deficiency* (2012) (68)
Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors. (2015) (67)
Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector. (2004) (66)
Enhancing rAAV vector expression in the lung (2005) (65)
Alternate Translation Initiation Codons Can Create Functional Forms of Cystic Fibrosis Transmembrane Conductance Regulator (*) (1995) (64)
Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis. (2007) (64)
Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method. (2011) (64)
Phase I Trial of Leber Congenital Amaurosis due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results (2008) (63)
Gene delivery in renal tubular epithelial cells using recombinant adeno-associated viral vectors. (2003) (61)
Adeno-associated virus: a ubiquitous commensal of mammals. (2005) (59)
Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector. (2005) (59)
In vivo post-transcriptional gene silencing of α-1 antitrypsin by adeno-associated virus vectors expressing siRNA (2007) (59)
Defective acid sphingomyelinase pathway with Pseudomonas aeruginosa infection in cystic fibrosis. (2009) (58)
Production of clinical-grade recombinant adeno-associated virus vectors. (2002) (57)
Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway. (2010) (57)
Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency (2017) (56)
CAR T-Cell Therapy: Progress and Prospects. (2017) (56)
Developing Core Competencies for the Prevention and Management of Prescription Drug Misuse: A Medical Education Collaboration in Massachusetts (2016) (54)
Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites. (2007) (53)
Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema (2018) (53)
Gene therapy for alpha-1 antitrypsin deficiency. (2011) (52)
Gene-Based Therapy for Alpha-1 Antitrypsin Deficiency (2013) (51)
Recombinant adeno-associated virus vectors for gene therapy (2004) (50)
In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency. (2018) (50)
Adeno-Associated Virus-Based Gene Therapy for Inherited Disorders (2005) (50)
Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaques. (2003) (49)
Recent developments in recombinant AAV-mediated gene therapy for lung diseases. (2005) (47)
A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis. (1998) (47)
A single-subunit NADH-quinone oxidoreductase renders resistance to mammalian nerve cells against complex I inhibition. (2003) (47)
Expression of a truncated cystic fibrosis transmembrane conductance regulator with an AAV5-pseudotyped vector in primates. (2007) (46)
Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency (2017) (45)
Effects of CFTR, interleukin-10, and Pseudomonas aeruginosa on gene expression profiles in a CF bronchial epithelial cell Line. (2004) (45)
Effect of Cigarette Smoke Exposure and Structural Modifications on the α-1 Antitrypsin Interaction with Caspases (2012) (44)
Murine Model for Cystic Fibrosis Bone Disease Demonstrates Osteopenia and Sex-Related Differences in Bone Formation (2009) (43)
Functional expression of the single subunit NADH dehydrogenase in mitochondria in vivo: a potential therapy for complex I deficiencies. (2004) (41)
Ex Vivo transduced liver progenitor cells as a platform for gene therapy in mice (2004) (41)
Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy. (2018) (40)
Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy. (2005) (40)
A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna. (2019) (40)
Recombinant adeno-associated virus-mediated global anterograde delivery of glial cell line-derived neurotrophic factor to the spinal cord: comparison of rubrospinal and corticospinal tracts in the rat. (2008) (39)
Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction. (2013) (39)
Accelerated Graduation and the Deployment of New Physicians During the COVID-19 Pandemic (2020) (38)
Asymptomatic Chlamydia trachomatis infections among sexually active men. (1986) (38)
Localized gene expression following administration of adeno-associated viral vectors via pancreatic ducts. (2005) (37)
Expression of the human multidrug resistance and glucocerebrosidase cDNAs from adeno-associated vectors: efficient promoter activity of AAV sequences and in vivo delivery via liposomes. (1996) (37)
Women’s Representation Among Members and Leaders of National Medical Specialty Societies (2019) (36)
Prospects for virus-based gene therapy for cystic fibrosis (1993) (36)
Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency (2017) (35)
Delayed Expression of Adeno-Associated Virus Vector DNA (1999) (35)
Methylphenidate Does Not Improve Cognitive Function in Healthy Sleep-Deprived Young Adults (2004) (34)
A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates (2018) (34)
Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector. (2002) (34)
Enhanced IgE allergic response to Aspergillus fumigatus in CFTR−/− mice (2006) (33)
Gene Therapy Vectors As Drug Delivery Systems (1995) (33)
Partial correction of the CFTR‐dependent ABPA mouse model with recombinant adeno‐associated virus gene transfer of truncated CFTR gene (2008) (33)
Benefits and complications of troleandomycin (TAO) in young children with steroid‐dependent asthma (1991) (32)
Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer (2018) (32)
Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials. (2015) (30)
Alveolar stem cell transduction by an adeno-associated viral vector. (1995) (30)
Current status of gene therapy for α-1 antitrypsin deficiency (2015) (29)
Cellular fusion for gene delivery to SCA1 affected Purkinje neurons (2011) (28)
Recombinant AAV Serotype and Capsid Mutant Comparison for Pulmonary Gene Transfer of α-1-Antitrypsin Using Invasive and Noninvasive Delivery. (2009) (28)
Adeno-associated virus vectors for gene therapy of cystic fibrosis. (1998) (28)
Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy. (2001) (27)
Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of alpha-1-antitrypsin using invasive and noninvasive delivery. (2009) (27)
Size does matter: overcoming the adeno-associated virus packaging limit (2000) (27)
In vivo gene therapy with adeno-associated virus vectors for cystic fibrosis. (1997) (26)
Training the next generation of biomedical investigators in glycosciences. (2016) (26)
Cystic Fibrosis Transmembrane Regulator Missing the First Four Transmembrane Segments Increases Wild Type and ΔF508 Processing * (2008) (26)
Development of adeno-associated virus vectors for gene therapy of cystic fibrosis. (1996) (26)
The rapidly evolving state of gene therapy (2018) (24)
Gene Transfer in Skeletal and Cardiac Muscle Using Recombinant Adeno‐Associated Virus (2013) (23)
Severe Toxicity in Nonhuman Primates and Piglets with Systemic High-Dose Administration of Adeno-Associated Virus Serotype 9-Like Vectors: Putting Patients First. (2018) (23)
Cystic Fibrosis Transmembrane Conductance Regulator Deficiency Exacerbates Islet Cell Dysfunction After β-Cell Injury (2006) (23)
Glucose‐Responsive Expression of the Human Insulin Promoter in HepG2 Human Hepatoma Cells (2003) (22)
Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy (2012) (21)
The pros and cons of immunomodulatory IL-10 gene therapy with recombinant AAV in a Cftr−/−-dependent allergy mouse model (2009) (20)
The murine α1-Proteinase inhibitor gene family: Polymorphism, chromosomal location, and structure (2002) (20)
The pyruvate dehydrogenase complex as a target for gene therapy. (2003) (20)
Pharmacokinetics and pharmacodynamic effects of nicotine nasal spray devices on cardiovascular and pulmonary function. (2000) (20)
Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans. (2016) (18)
CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing (2021) (18)
AAV gene therapy for Tay-Sachs disease (2022) (18)
Teaching hypothesis-oriented thinking to medical students: the University of Florida's clinical investigation program. (2001) (18)
Recombinant Adeno-Associated Virus Gene Therapy for Cystic Fibrosis and α1-Antitrypsin Deficiency (2002) (17)
Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans (2020) (17)
Gene Therapy for Cystic Fibrosis (2008) (17)
No Immune Responses by the Expression of the Yeast Ndi1 Protein in Rats (2011) (17)
101. Toxicology and Biodistribution Studies of a Recombinant Adeno-Associated Virus 2 (rAAV2) Alpha-1 Antitrypsin (AAT) Vector|[ast]| (2004) (17)
Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy. (2013) (17)
Induction of group IVC phospholipase A2 in allergic asthma: transcriptional regulation by TNFα in bronchoepithelial cells. (2012) (16)
Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle. (2016) (16)
Gene therapy for pyruvate dehydrogenase E1alpha deficiency using recombinant adeno-associated virus 2 (rAAV2) vectors. (2002) (16)
Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome. (2012) (16)
Systemic correction of a fatty acid oxidation defect by intramuscular injection of a recombinant adeno-associated virus vector. (2005) (15)
CFTR mutations impart elevated immune reactivity in a murine model of cystic fibrosis related diabetes. (2008) (15)
Recurrent Pneumonia in Children: A Case Report and Approach to Diagnosis (2006) (15)
Adeno-associated virus transduction of islets with interleukin-4 results in impaired metabolic function in syngeneic marginal islet mass transplantation. (2002) (14)
Empty Adeno-Associated Virus Capsids: Contaminant or Natural Decoy? (2017) (14)
Gene Transfer in the Lung Using Recombinant Adeno‐Associated Virus (2012) (13)
Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors. (2005) (13)
Dietl syndrome: intermittent ureteropelvic junction obstruction as a cause of episodic abdominal pain. (1988) (13)
Charting a clear path: the ASGCT Standardized Pathways Conference. (2014) (12)
Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases (2013) (12)
Recombinant adeno-associated virus gene therapy for cystic fibrosis and alpha(1)-antitrypsin deficiency. (2002) (12)
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells (2021) (12)
Revisiting the "New" Inflammatory Toxicities of Adeno-Associated Virus Vectors. (2020) (12)
Therapeutic Germ Line Alteration: Has CRISPR/Cas9 Technology Forced the Question? (2015) (11)
Modulation of exaggerated-IgE allergic responses by gene transfer-mediated antagonism of IL-13 and IL-17e. (2010) (11)
Adeno-associated virus-mediated gene transfer for lung diseases. (2005) (11)
Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency. (2015) (11)
The murine alpha(1)-proteinase inhibitor gene family: polymorphism, chromosomal location, and structure. (2002) (11)
The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency. (2007) (10)
Recent developments in the protection of pediatric research subjects. (2006) (10)
Biochemical correction of short-chain acyl-coenzyme A dehydrogenase deficiency after portal vein injection of rAAV8-SCAD. (2008) (10)
N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearance. (2011) (9)
In vitro and in vivo Functional Characterization of Gutless Recombinant SV40-derived CFTR Vectors (2009) (9)
Restrictive Lung Disease in the Cu/Zn Superoxide-Dismutase 1 G93A Amyotrophic Lateral Sclerosis Mouse Model. (2017) (8)
Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees. (2008) (8)
Towards a rAAV-based gene therapy for ADA-SCID: from ADA deficiency to current and future treatment strategies. (2008) (8)
Large-scale molecular epidemiological analysis of AAV in a cancer patient population (2021) (8)
Autoimmunity in a genetic disease—a cautionary tale. (2010) (8)
Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups. (2017) (7)
Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency. (2017) (7)
Hepatitis Virus Protein X-Phenylalanine Hydroxylase fusion proteins identified in PKU mice treated with AAV-WPRE vectors Research Article (2008) (7)
AAV Is Now a Medicine: We Had Better Get This Right. (2017) (7)
Recombinant adeno‐associated virus‐mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD‐deficient mice (2008) (6)
Effect of allergy and inflammation on eicosanoid gene expression in CFTR deficiency. (2013) (6)
Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study (2019) (6)
Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy. (2012) (6)
Prime Editing: A Novel Cas9-Reverse Transcriptase Fusion May Revolutionize Genome Editing. (2019) (6)
Ethical Implications of the Cost of Molecularly Targeted Therapies. (2015) (6)
216. Long Term Portal Vein Administration of AAV-WPRE Vector Results in Increased Incidence of Neoplastic Disease and Hepatic Pathology (2006) (6)
Gene Therapy: Use of Viruses as Vectors (2008) (6)
Endocrine parameters of cystic fibrosis: Back to basics (2009) (5)
An Antiapoptotic Role for α1-Antitrypsin in the Prevention of Emphysema (2006) (5)
Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency. (2011) (5)
AAV9 gene replacement therapy for respiratory insufficiency in very‐long chain acyl‐CoA dehydrogenase deficiency (2019) (5)
INHERITED DISEASE RESEARCH ARTICLE Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients (2000) (5)
Common pathways to Dean of Medicine at U.S. medical schools (2021) (5)
The Science Policy Implications of a Trump Presidency. (2017) (5)
GENE-BASED THERAPEUTICS FOR RARE GENETIC NEURODEVELOPMENTAL PSYCHIATRIC DISORDERS. (2022) (4)
Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods. (2015) (4)
DNA Vaccination in 2018: An Update. (2018) (4)
Manufacture and Stability of the Recombinant Adeno-Associated Virus Serotype 2 Vector Reference Standard (2008) (4)
Adeno‐associated viral vectors for gene therapy (2005) (4)
Assuring Integrity in the Residency Match Process (2019) (4)
Gene therapy for cystic fibrosis. (2001) (4)
In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno‐associated virus (rAAV) serotypes 2 and 8 (2007) (4)
Leber Congenital Amaurosis gene therapy clinical trial (2009) (3)
In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis. (2021) (3)
The role of gene and cell therapy in the era of health care reform. (2011) (3)
Delivery of CFTR gene to rabbit airways by gelatin-DNA microspheres (1996) (3)
Combination of drug and gene delivery by gelatin nanospheres for the treatment of cystic fibrosis (1997) (3)
Alpha-1 Antitrypsin Deficiency as a Candidate for Gene Editing. (2018) (3)
Preclinical study design for rAAV. (2011) (3)
Volume and Infusion Rate Dynamics of Intraparenchymal CNS Infusion in a Large Animal Model. (2020) (3)
Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice (2022) (3)
2020: Gene Therapy Enters Its Fourth Decade. (2020) (3)
Gene Therapy for Rare Neurological Disorders (2022) (3)
Guiding the Future of Medical Education through “Enlightened” Accreditation Policy (2015) (2)
Two-Plasmid Packaging System for Recombinant Adeno-Associated Virus (2020) (2)
6 Adeno-Associated Viral Vectors (2001) (2)
24. Sustained Expression with Partial Correction of Neutrophil Defects 5 Years After Intramuscular rAAV1 Gene Therapy for Alpha-1 Antitrypsin Deficiency (2016) (2)
Therapeutic Advances in Germany and Beyond. (2017) (2)
Adeno-associated viral vectors for CF gene therapy. (2002) (2)
First-in-human AAV Gene Therapy for Tay-Sachs Disease (2021) (2)
Top Five Gene Therapy Stories of 2019. (2019) (2)
The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute. (2017) (2)
What is suppression of anti-adeno-associated virus capsid T-cells achieving? (2014) (2)
Adeno-Associated Virus and Other New DNA Virus Vectors (1997) (1)
Pharmacokinetics and pharmacodynamics of a nicotine nasal spray (1998) (1)
Swinging for the fences: persistent and efficient liver-directed gene therapy for hemophilia A (2004) (1)
AN IMPROVED METHOD OF RIBAVIRIN ADMINISTRATION TO THE NONINTUBATED INFANT (1994) (1)
Gene Drives: Biological Shield or Ecological Menace? (2016) (1)
Treatment of Patients with Leber Congenital Amaurosis Type 2 with an AAV Vector Expressing RPE65 (2013) (1)
Regulatory pathway on the manufacture and quality control of recombinant adeno-associated virus vectors (2018) (1)
Engraftment of Human Hepatocytes in the PiZ-NSG Mouse Model. (2020) (1)
Moving Forward after Two Deaths in a Gene Therapy Trial of Myotubular Myopathy (2020) (1)
Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA. (2017) (1)
European Society of Gene and Cell Therapy (ESGCT) at 25: A Gene Therapy Community at Its Prime and on the Move. (2017) (1)
601. Gene Therapy for Cockayne Syndrome (2015) (1)
CRISPR Keeps Things Fresh: Next-Generation Tools for Gene Editing. (2021) (1)
Correction for Desplats et al., Inclusion formation and neuronal cell death through neuron-to-neuron transmission of α-synuclein (2009) (1)
644. Gene Delivery for Chronic Pain Control: Micrcroencapsulated Plasmid DNA Encoding the Anti-Inflammatory Cytokine Gene, Interleukin-10 (IL-10) (2005) (1)
Moving forward toward a cure for Parkinson's: neuropathology of the nigrostriatal pathway determines the location of growth factor delivery. (2011) (1)
Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy. (2018) (1)
Supporting Families Considering Participation in a Clinical Trial: Parent-Provider Perspectives (2021) (1)
Muscle-Directed Gene Therapy for Alpha-1 Antitrypsin Deficiency (2019) (1)
Transcription Activator-Like Nucleases Enable Allogeneic Chimeric Antigen Receptor-T Cell Therapy in Humans. (2015) (1)
Alternative capsid strategies for targeting recombinant AAV gene therapy (2003) (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)
The Role of Patient Advocacy Organizations in Advancing Human Gene Therapy. (2015) (1)
Codon Optimization for Alpha 1-Antitrypsin Disease (2012) (0)
Adeno-Associated Virus (AAV)-Based Gene Therapy for Defects in Oxidative Metabolism (1999) (0)
ESGCT 2021: Virtually Pan-European. (2021) (0)
Clinical Evaluation of a Recombinant Adeno-Associated Virus (rAAV) Alpha-1 Antitrypsin (AAT) Gene Therapy Vector. (2009) (0)
Recombinant AAV vectors for gene transfer to the lung: a compartmental approach (2005) (0)
Black Lives Matter to Gene Therapy. (2020) (0)
What the Gene Therapy Community Should Do About Sexual Harassment. (2019) (0)
Gene Therapy for Endocrine Disorders: A Promising Intervention. (2022) (0)
Cystic Fibrosis: Gene Therapy (2006) (0)
GENE THERAPY (2019) (0)
Why Human Gene Therapy Scientists Should Care About Model Organisms. (2016) (0)
498. AAV ?264CFTR Enhances Maturation of ?F508CFTR and wt CFTR Expression (2006) (0)
The signal and the trap: targeted delivery and retention of proteins in the mitochondrion. (2003) (0)
The End of the Beginning of Gene Therapy. (2015) (0)
Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia. (2021) (0)
Adeno-Associated Virus-Human Bocavirus 1 Chimeric Vectors: Ferreting Out Their Role in Airway Gene Therapy. (2017) (0)
Immune Responses to Recombinant Adenoviruses As Gene Therapy Vectors and COVID-19 Vaccines: A Two-Edged Sword. (2021) (0)
111. Post-Transcriptional Gene Silencing of Alpha-1 Antitrypsin by Small Interfering RNAs (siRNA) (2006) (0)
ADENO-ASSOCIATED VIRUS TRANSDUCTION OF ISLETS WITH INTERLEUKIN-4 RESULTS IN IMPAIRED METABOLIC FUNCTION IN SYNGENEIC MARGINAL ISLET MASS TRANSPLANTATION 1 (2018) (0)
Gene Therapy for Alcoholism and Other Substance Use Disorders. (2017) (0)
Women Lead Gene Therapy Science in 2021. (2021) (0)
Gene therapy for alpha-1 antitrypsin deficiency: an update (2023) (0)
New Horizons for Immune Gene Therapy. (2019) (0)
Evolution of the Alpha-1 Antitrypsin Muscle Gene Therapy: Translation from Clinical Trial to Benchtop and Back Again (2017) (0)
Epigenome Editing Strategies for Low Back Pain. (2019) (0)
Writing the Story of Gene Editing and CRISPR: An Interview with Kevin Davies, PhD. (2020) (0)
Interleukin-4 Expressed from Adeno-Assciated Virus Transduced Islet Grafts Fails To Improve the Reversal of Hyperglycemia in Syngeneic and Allogeneic Islet Transplantation Models (2002) (0)
Serum Levels of Alpha-1 Antitrypsin following Vascular Limb or Intra-Muscular Delivery of AAV1 or AAV8 Gene Therapy Vectors in Rhesus Macaques (2016) (0)
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient. (2022) (0)
Liver targeting with rAAV7: balancing tropism with immune profiles (2021) (0)
Gene and Cell Therapy in 2018: A Look Ahead. (2018) (0)
Diagnostic and therapeutic applications of bacteriophage and adeno-associated virus technologies in pulmonary emphysema (2006) (0)
881. Safety, Efficacy and Biodistribution of Recombinant AAV2-RPE65 Vector Delivered by Ocular Subretinal Injection (2006) (0)
Getting Tough on Capsid Screening: Tough Decoys Enable Barcoding of Vectors Capable of both Entry and Expression. (2019) (0)
Response to A. de Grey (2001) (0)
Analyzing clinical observations to better understand and manage immune responses to AAV gene therapies. (2023) (0)
Precision of Exon Skipping with U7 Constructs. (2021) (0)
Interfacing Stem Cells with Gene Therapy (2010) (0)
Asymptomatic Chlamydia Trachomatis Infections Among Sexually Active Men (1987) (0)
Defining Scholarship for Today and Tomorrow. (2022) (0)
In Reply to Ramotshwana et al. (2021) (0)
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. (2023) (0)
A Message to Our Community in the Midst of the COVID-19 Pandemic. (2020) (0)
Ringing in the Changes. (2020) (0)
Procurement of State-of-the-Art Research Equipment to Support Faculty Members Within the RNAi Therapeutics Institute (2010) (0)
Senior Gene Therapy Scientists Take a Stand Against Human Embryo Gene Editing. (2019) (0)
A Gene Therapy Scientist's Life Well-Lived. (2016) (0)
rAAV gene transfer to the liver (2005) (0)
377. Expression of Acid |[alpha]|-Glucosidase in Nonhuman Primate Diaphragm and Peritoneum after Direct In Utero Delivery of Recombinant Adeno-Associated Virus Type 1 (2004) (0)
Secretion of functional α1-antitrypsin is cell type dependent: Implications for intramuscular delivery for gene therapy (2022) (0)
The Year in Review: The Top Five Papers of 2018. (2018) (0)
183. Developing Strategies To Improve the Current Clinical Vector AAV1-CB-AAT for Alpha-One Antitrypsin Deficiency (2015) (0)
1055. Anterograde Delivery of Gene Product by rAAV5 Vectors|[ast]| (2004) (0)
Precision-guided genetic ordnance: new developments in receptor targeting of recombinant AAV (2003) (0)
Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer (2023) (0)
Sleeping Beauty Awakens New Interest. (2017) (0)
Gene Therapy Untangles the Problem of Chronic Traumatic Encephalopathy. (2020) (0)
The Negative Effects of Immigration Restrictions on the Gene Therapy Community. (2017) (0)
In Reply to Signer and Curtin and to Goyal et al. (2020) (0)
667. Effects of Various Physical, Chemical, and Biological Exposures on rAAV Stability and Function (2015) (0)
ESGCT 2022: The New Normal for the Gene Therapy Community. (2022) (0)
875. Ribozyme Approaches towards Down-Regulation of Pi|[ast]|Z Mutant Human A-1 Anti-Trypsin (2004) (0)
Free Gene and Cell Therapy Services Offered by the National Heart, Lung, and Blood Institute of the National Institutes of Health. (2020) (0)
Pushing the envelope in the lung (2011) (0)
A Phase I Study of AAV-CFTR Administration in Adult Cystic Fibrosis Patients 1794 (1997) (0)
Recombinant Adeno-Associated Virus-Based Gene Therapy for Disorders Detected by Newborn Screening: Inherent Limitations of This Approach. (2018) (0)
Gene and Cell Therapy for Inherited and Acquired Immune Deficiency. (2021) (0)
702. Detection and Isolation of Immune Cells After Intramuscular AAV Injection in Mouse and Human (2016) (0)
A Brief Guide to Gene Therapy Treatments for Pulmonary Diseases (2010) (0)
Phase 2 Clinical Trial Of A Recombinant Adeno-Associated Virus (RAAV) Alpha-1 Antitrypsin (AAT) Gene Therapy Vector (2011) (0)
1077. Phase I Clinical Trial of Recombinant Adeno-Associated Virus (rAAV)-Alpha-1 Antitrypsin Vectors (2005) (0)
The Renaissance of Gene and Cell Therapy: Florence 2016. (2016) (0)
NEW RECOMBINANT ADENO-ASSOCIATED VIRUS (rAAV)-BASED THERAPEUTICS REACH PIVOTAL PHASE 2 CLINICAL TRIALS (2011) (0)
Hepatic Changes Associated with Chronic Alcohol Exposure in an Alpha-1 Antitrypsin PiZ Mouse Model (2017) (0)
Barrie J. Carter: The Science and the Scientist. (2020) (0)
In Reply to Manion and Khan. (2017) (0)
115. In Vivo Expression of Human Cob(I)alamin Adenosyltransferase Using rAAV Serotypes 1,2 and 8, an Approach to Gene Therapy for Methylmalonic Aciduria (MMA)|[ast]| (2005) (0)
Production de virions de virus adeno-associe (aav) recombinants pseudo-types (2003) (0)
728. Safety and Bioactivity of rAAV2-hAAT in alpha-1 Antitrypsin-Deficient Patients in a Phase I Clinical Trial (2006) (0)
Science Policy and Funding Implications of the 2020 U.S. Election. (2020) (0)
DMD & BMD – CLINICAL P.43 Patient and family centric care for Duchenne and Becker muscular dystrophy: towards the IPU (Integrated Practice Unit) model (2020) (0)
98. Adeno-Associated Virus SerOtype 8 as a Vector for HEPATIC Delivery of alpha-1 Anti-Trypsin (AAT) (2004) (0)
183. Elucidation of Alpha-1 Antitrypsin Levels in Human Infant Blood Samples (2016) (0)
Special Note from the Editor-in-Chief. (2016) (0)
705. Selective Advantage of Hepatocytes Expressing Wild-Type Alpha-1 Antitrypsin (AAT) in a Novel Human Liver Xenograft Model: A Model for Correction of a Gain-Of-Function Mutation (2015) (0)
Base Editing to the Rescue. (2021) (0)
1083. Systemic rAAV-CB-IL-10 Expression and Its Long Term Effects on CD4+CD25+ T Cell Populations (2005) (0)
The Challenge of Maintaining our Physician-Scientist Workforce (2016) (0)
Impact on Women's Health: Gene Therapy in Gynecologic Oncology. (2020) (0)
It Is Time to Call Anti-Asian Bias What It Is: Racism. (2021) (0)
654. Quantifying GDNF Levels in the Spinal Cord after Intracerebral Delivery of rAAV5 GDNF (2005) (0)
The Target's the Thing. (2016) (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)
Gene Therapy 2016: The Pipeline Is Swelling (2016) (0)
rAAV-based compositions and methods (2016) (0)
One More Controversy: Adeno-Associated Virus in Stem Cells. (2017) (0)
Production of the high titers of recombinant AAV vectors (1994) (0)
169. Intrapancreatic injections of adeno-associated virus vectors for local gene expression (2004) (0)
CHAPTER 44 – Gene Transfer in the Lung (2008) (0)
106. Functional Human alpha 1 Antitrypsin Secreted from Muscle Transduced by Adeno-Associated Virus (rAAV1) Vector|[ast]| (2004) (0)
viral vector recombinant adeno-associated encoding alpha-1 antitrypsin gene therapy. (1999) (0)
In utero efficacy of cystic fibrosis gene therapy: difficult studies, positive or negative. (2008) (0)
The attainment of high titers of recombinant AAV vector (1994) (0)
436. Recombinant Adeno-Associated Virus Gene Therapy for Cob(I)Alamin Adenosyltransferase Deficiency|[ast]| (2004) (0)
Recombinant adeno-associated virus vector encoding the alpha-1-antitrypsin in gene therapy (1999) (0)
493. Enhanced Transgene Transfer and Expression by an Endobronchially Delivered Pseudotyped AAV2/5 Vector (2004) (0)
therapy for hemophilia A Swinging for the fences: persistent and efficient liver-directed gene (2013) (0)
Gene and Cell Therapy in China: Highlighting Excellence in the 21st Century ： 21. (2018) (0)
Cell Injury , Repair , Aging and Apoptosis-1 Antitrypsin Inhibits Caspase-3 Activity , Preventing Lung Endothelial Cell Apoptosis (2006) (0)

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University of Florida
University of Massachusetts Medical School
Johns Hopkins University
University of Massachusetts

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