Luigi Naldini

Q: What Schools Are Affiliated With Luigi Naldini

Luigi Naldini is affiliated with the following schools: University of Antwerp, University of Kentucky, Vita-Salute San Raffaele University, University of Siena, University of Washington, University of California, San Francisco, University of Turin, Sapienza University of Rome

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Luigi Naldini

Philosophy

#3640

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#5849

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#1557

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#2339

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Luigi Naldini

Biology

#4594

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#6831

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Virology

#94

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#97

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Molecular Biology

#421

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#430

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Philosophy
Biology

Luigi Naldini's Degrees

Doctorate Medicine University of Turin
PhD Molecular Medicine University of Turin

Why Is Luigi Naldini Influential?

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According to Wikipedia, Luigi Naldini is an Italian gene therapist, professor of cell and tissue biology and of gene and cell therapy at Vita-Salute San Raffaele University, and the director of San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Often considered as "the father of the lentivirus gene therapy", he is globally known as the pioneer of "the development and applications of lentiviral vectors for gene transfer" that allowed the gene therapy treatment of several genetic diseases. He is also a scientific co-founder of three biotech start-up companies, Genenta in Milan and New York, Chroma Medicine in Milan and Boston, Genespire in Milan. He is also the former president of European Society of Gene and Cell Therapy and an elected member of European Molecular Biology Organization . In 2019, he was awarded Louis-Jeantet Prize for Medicine "for taking gene therapy from the bench to the bedside".

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Luigi Naldini'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

In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector (1996) (4939)
A Third-Generation Lentivirus Vector with a Conditional Packaging System (1998) (2975)
Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo (1997) (2034)
Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery (1998) (1853)
Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. (1996) (1600)
Hepatocyte growth factor is a potent angiogenic factor which stimulates endothelial cell motility and growth (1992) (1350)
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics (2001) (1332)
Tie2 identifies a hematopoietic lineage of proangiogenic monocytes required for tumor vessel formation and a mesenchymal population of pericyte progenitors. (2005) (1304)
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy (2013) (1030)
Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences (2000) (981)
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery (2007) (888)
Gene therapy returns to centre stage (2015) (885)
Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome (2013) (812)
Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector (1997) (744)
Hepatocyte growth factor (HGF) stimulates the tyrosine kinase activity of the receptor encoded by the proto-oncogene c-MET. (1991) (710)
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration (2006) (696)
Scatter factor and hepatocyte growth factor are indistinguishable ligands for the MET receptor. (1991) (660)
Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells (2003) (602)
Extracellular proteolytic cleavage by urokinase is required for activation of hepatocyte growth factor/scatter factor. (1992) (587)
Targeting the ANG2/TIE2 axis inhibits tumor growth and metastasis by impairing angiogenesis and disabling rebounds of proangiogenic myeloid cells. (2011) (573)
Cleavage of the Plasma Membrane Na+/Ca2+ Exchanger in Excitotoxicity (2005) (556)
The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. (2009) (552)
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state (2007) (546)
An unbiased genome-wide analysis of zinc-finger nuclease specificity (2011) (533)
Targeted Genome Editing in Human Repopulating Hematopoietic Stem Cells (2014) (524)
FcRgamma activation regulates inflammation-associated squamous carcinogenesis. (2010) (501)
Identification of proangiogenic TIE2-expressing monocytes (TEMs) in human peripheral blood and cancer. (2007) (496)
Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer (2006) (494)
Forebrain ependymal cells are Notch-dependent and generate neuroblasts and astrocytes after stroke (2009) (438)
Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors. (1999) (436)
A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR. (2012) (434)
Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer (2012) (424)
Cardiomyocytes induce endothelial cells to trans-differentiate into cardiac muscle: Implications for myocardium regeneration (2001) (399)
Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications (2009) (391)
Lentiviral vectors: excellent tools for experimental gene transfer and promising candidates for gene therapy (2000) (354)
A distinguishing gene signature shared by tumor-infiltrating Tie2-expressing monocytes, blood "resident" monocytes, and embryonic macrophages suggests common functions and developmental relationships. (2009) (352)
Ex vivo gene transfer and correction for cell-based therapies (2011) (350)
Efficient lentiviral transduction of liver requires cell cycling in vivo (2000) (336)
Inheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic Editing (2016) (329)
Cell-substratum interaction of cultured avian osteoclasts is mediated by specific adhesion structures (1984) (324)
Stable knockdown of microRNA in vivo by lentiviral vectors (2009) (324)
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial (2016) (322)
Lentiviruses as gene transfer agents for delivery to non-dividing cells. (1998) (318)
RON is a heterodimeric tyrosine kinase receptor activated by the HGF homologue MSP. (1994) (313)
Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters (2005) (312)
Site-specific integration and tailoring of cassette design for sustainable gene transfer (2011) (303)
Targeting the tumor and its microenvironment by a dual-function decoy Met receptor. (2004) (301)
Tie2-expressing monocytes: regulation of tumor angiogenesis and therapeutic implications. (2007) (277)
CD44v6-targeted T cells mediate potent antitumor effects against acute myeloid leukemia and multiple myeloma. (2013) (276)
Adopt a moratorium on heritable genome editing (2019) (274)
Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. (2004) (273)
Biological Activation of pro-HGF (Hepatocyte Growth Factor) by Urokinase Is Controlled by a Stoichiometric Reaction (*) (1995) (261)
Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. (2002) (259)
Tie2-expressing monocytes and tumor angiogenesis: regulation by hypoxia and angiopoietin-2. (2007) (255)
Tumor-targeted interferon-alpha delivery by Tie2-expressing monocytes inhibits tumor growth and metastasis. (2008) (254)
The MET oncogene drives a genetic programme linking cancer to haemostasis (2005) (254)
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. (2007) (251)
Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. (2002) (251)
ERK1 and ERK2 mitogen-activated protein kinases affect Ras-dependent cell signaling differentially (2006) (232)
Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. (2004) (225)
Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection. (2011) (225)
A functional domain in the heavy chain of scatter factor/hepatocyte growth factor binds the c-Met receptor and induces cell dissociation but not mitogenesis. (1992) (225)
TIE2-expressing macrophages limit the therapeutic efficacy of the vascular-disrupting agent combretastatin A4 phosphate in mice. (2011) (220)
Generation of HIV-1 derived lentiviral vectors. (2002) (216)
Tyrosines1234-1235 are critical for activation of the tyrosine kinase encoded by the MET proto-oncogene (HGF receptor). (1994) (214)
Generation of potent and stable human CD4+ T regulatory cells by activation-independent expression of FOXP3. (2008) (212)
Transduction of human CD34+ CD38- bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors. (2000) (200)
In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice (2001) (199)
Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. (2006) (196)
miR-511-3p modulates genetic programs of tumor-associated macrophages. (2012) (194)
In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance. (2007) (193)
Attenuation of miR-126 Activity Expands HSC In Vivo without Exhaustion (2012) (192)
Identification of Hematopoietic Stem Cell–Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy (2010) (192)
Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products (2017) (186)
The tyrosine kinase encoded by the MET proto-oncogene is activated by autophosphorylation (1991) (177)
Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors. (2004) (175)
Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. (2005) (174)
Comprehensive genomic access to vector integration in clinical gene therapy (2009) (173)
Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response (2019) (171)
Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application. (2011) (170)
The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity (2012) (169)
In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases (2016) (168)
A new-generation stable inducible packaging cell line for lentiviral vectors. (2001) (161)
Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. (2010) (160)
Tie2 identifies a hematopoietic monocytes required for tumor lineage of proangiogenic vessel formation and a mesenchymal population of pericyte progenitors (2005) (158)
Human T lymphocytes transduced by lentiviral vectors in the absence of TCR activation maintain an intact immune competence. (2003) (158)
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia (2019) (156)
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1 (2017) (156)
Stability of lentiviral vector-mediated transgene expression in the brain in the presence of systemic antivector immune responses. (2005) (156)
HIV-based vectors. Preparation and use. (2002) (155)
Elusive identities and overlapping phenotypes of proangiogenic myeloid cells in tumors. (2010) (155)
Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors. (2002) (154)
Genomic instability in induced stem cells (2011) (153)
Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation (2012) (150)
Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo. (2014) (143)
Hepatocyte-Targeted Expression by Integrase-Defective Lentiviral Vectors Induces Antigen-Specific Tolerance in Mice with Low Genotoxic Risk (2011) (141)
In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance. (2009) (141)
miR-126 Regulates Distinct Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells (2016) (135)
Interaction of Human Immunodeficiency Virus-Derived Vectors with Wild-Type Virus in Transduced Cells (1999) (130)
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study (2019) (125)
miR-142-3p prevents macrophage differentiation during cancer-induced myelopoiesis. (2013) (124)
CRISPR germline engineering—the community speaks (2015) (124)
Role of haematopoietic cells and endothelial progenitors in tumour angiogenesis. (2006) (121)
‘Advanced’ generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo (2003) (121)
Identification of the major autophosphorylation site of the Met/hepatocyte growth factor receptor tyrosine kinase. (1991) (120)
Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy (2017) (119)
Pseudotyped human lentiviral vector-mediated gene transfer to airway epithelia in vivo (2000) (119)
MET overexpression turns human primary osteoblasts into osteosarcomas. (2006) (119)
Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction. (2004) (118)
Detection of phosphotyrosine‐containing proteins in the detergent‐insoluble fraction of RSV‐transformed fibroblasts by azobenzene phosphonate antibodies. (1984) (116)
Dynamics and genomic landscape of CD8+ T cells undergoing hepatic priming (2019) (115)
Systemic and Targeted Delivery of Semaphorin 3A Inhibits Tumor Angiogenesis and Progression in Mouse Tumor Models (2011) (114)
CD4+ T Cells from IPEX Patients Convert into Functional and Stable Regulatory T Cells by FOXP3 Gene Transfer (2013) (114)
Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations. (2012) (111)
Liver-directed lentiviral gene therapy in a dog model of hemophilia B (2015) (111)
In vivo targeting of tumor endothelial cells by systemic delivery of lentiviral vectors. (2003) (106)
Tie2-expressing monocytes (TEMs): novel targets and vehicles of anticancer therapy? (2009) (103)
miR-126 Regulates Distinct Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells (2016) (102)
An uncleavable form of pro-scatter factor suppresses tumor growth and dissemination in mice. (2004) (101)
Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells. (2002) (100)
A role for miR-155 in enabling tumor-infiltrating innate immune cells to mount effective antitumor responses in mice. (2013) (99)
TIE2-expressing monocytes/macrophages regulate revascularization of the ischemic limb (2013) (98)
Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I. (2011) (96)
Applications of gene therapy to the CNS. (1996) (96)
Loss of transcriptional control over endogenous retroelements during reprogramming to pluripotency (2014) (94)
Lentiviral vectors, two decades later (2016) (94)
Angiopoietin-2 TIEs Up Macrophages in Tumor Angiogenesis (2011) (91)
Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models. (2009) (90)
Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient. (2002) (90)
Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice. (2012) (90)
The impact of amino acid variability on alloreactivity defines a functional distance predictive of permissive HLA-DPB1 mismatches in hematopoietic stem cell transplantation. (2015) (89)
HIV-1-derived lentiviral vectors. (2002) (88)
Regulated and multiple miRNA and siRNA delivery into primary cells by a lentiviral platform. (2009) (88)
Genetic Engineering of Hematopoiesis for Targeted IFN-α Delivery Inhibits Breast Cancer Progression (2014) (87)
Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects. (2006) (85)
Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation. (2006) (84)
Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer (2013) (82)
ISSCR Guidelines for Stem Cell Research and Clinical Translation: The 2021 update (2021) (82)
Transduction of a gene expression cassette using advanced generation lentiviral vectors. (2002) (81)
Osteoclasts and monocytes have similar cytoskeletal structures and adhesion property in vitro. (1983) (80)
Hepatocyte Growth Factor Is a Regulator of Monocyte-Macrophage Function1 (2001) (80)
Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction. (2006) (78)
Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking (2020) (77)
A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A. (2011) (76)
Targeted gene therapy and cell reprogramming in Fanconi anemia (2014) (76)
Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy. (2009) (74)
Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells (2018) (74)
Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives (2019) (73)
Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector. (2005) (73)
Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models. (2010) (73)
A Comeback for Gene Therapy (2009) (69)
SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies. (2007) (69)
Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome (2012) (69)
Efficient Tet-dependent expression of human factor IX in vivo by a new self-regulating lentiviral vector. (2005) (65)
Phosphotyrosine antibodies identify the p210c-abl tyrosine kinase and proteins phosphorylated on tyrosine in human chronic myelogenous leukemia cells (1986) (64)
Neural Stem Cell Gene Therapy Ameliorates Pathology and Function in a Mouse Model of Globoid Cell Leukodystrophy (2011) (64)
Lentivirus-mediated gene transfer into hematopoietic repopulating cells in baboons (2002) (64)
Post-natal cardiomyocytes can generate iPS cells with an enhanced capacity toward cardiomyogenic re-differentation (2012) (62)
Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis. (2015) (61)
Lentiviral vector integration profiles differ in rodent postmitotic tissues. (2011) (61)
Reprogramming T lymphocytes for melanoma adoptive immunotherapy by T-cell receptor gene transfer with lentiviral vectors. (2009) (61)
Transcriptional Targeting of Lentiviral Vectors by Long Terminal Repeat Enhancer Replacement (2002) (60)
A human immunodeficiency virus type 1 pol gene-derived sequence (cPPT/CTS) increases the efficiency of transduction of human nondividing monocytes and T lymphocytes by lentiviral vectors. (2002) (59)
Molecular characteristics of HGF-SF and its role in cell motility and invasion. (1993) (58)
Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy (2014) (58)
Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery. (2005) (58)
Receptor for bombesin with associated tyrosine kinase activity. (1986) (56)
NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease. (2017) (56)
Development of lentiviral vectors for antiangiogenic gene delivery (2001) (56)
Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector. (2005) (55)
Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination. (2013) (54)
Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice (2013) (54)
Efficiency of onco-retroviral and lentiviral gene transfer into primary mouse and human B-lymphocytes is pseudotype dependent. (2003) (53)
Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy. (2006) (53)
Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy. (2007) (53)
FcR g Activation Regulates Inflammation-Associated Squamous Carcinogenesis (2011) (53)
Intracellular distribution of nerve growth factor in rat pheochromocytoma PC12 cells: evidence for a perinuclear and intranuclear location. (1980) (52)
Cyclosporin a and rapamycin relieve distinct lentiviral restriction blocks in hematopoietic stem and progenitor cells. (2015) (52)
Insulin B chain 9–23 gene transfer to hepatocytes protects from type 1 diabetes by inducing Ag-specific FoxP3+ Tregs (2015) (52)
Lentiviral gene transfer ameliorates disease progression in Long-Evans cinnamon rats: An animal model for Wilson disease (2006) (50)
Tracking differentiating neural progenitors in pluripotent cultures using microRNA-regulated lentiviral vectors (2010) (50)
Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. (2012) (50)
Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients (2017) (49)
miRNA-126 Orchestrates an Oncogenic Program in B Cell Precursor Acute Lymphoblastic Leukemia. (2016) (49)
Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates (2019) (48)
Intracellular calcium regulates the tyrosine kinase receptor encoded by the MET oncogene. (1991) (48)
Hepatocyte growth factor and its receptor, the tyrosine kinase encoded by the c-MET proto-oncogene. (1994) (48)
B-cell reconstitution after lentiviral vector–mediated gene therapy in patients with Wiskott-Aldrich syndrome (2015) (48)
Forkhead box protein 3 (FOXP3) mutations lead to increased TH17 cell numbers and regulatory T-cell instability. (2011) (47)
The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche. (2010) (47)
Oncoretroviral and lentiviral vector-mediated gene therapy. (2002) (46)
Lentiviral Vector Gene Transfer Is Limited by the Proteasome at Postentry Steps in Various Types of Stem Cells (2008) (46)
Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting. (2011) (45)
Gene therapy of storage disorders by retroviral and lentiviral vectors. (2005) (44)
Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe‐affected non‐human primates by intracerebral lentiviral gene therapy (2016) (44)
HIV-1-derived lentiviral vectors directly activate plasmacytoid dendritic cells, which in turn induce the maturation of myeloid dendritic cells. (2011) (43)
Lentiviral vectors escape innate sensing but trigger p53 in human hematopoietic stem and progenitor cells (2017) (43)
Molecular evidence of inefficient transduction of proliferating human B lymphocytes by VSV-pseudotyped HIV-1-derived lentivectors. (2004) (41)
Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome. (2011) (39)
In vivo phosphorylation and dephosphorylation of the platelet-derived growth factor receptor studied by immunoblot analysis with phosphotyrosine antibodies. (1986) (39)
Design and optimization of lentiviral vectors for transfer of GALC expression in Twitcher brain (2006) (38)
The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4+CD25+ regulatory T cells. (2007) (38)
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access (2022) (38)
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. (2021) (36)
Genome editing for scalable production of alloantigen‐free lentiviral vectors for in vivo gene therapy (2017) (36)
Activation of the protein-tyrosine kinase associated with the bombesin receptor complex in small cell lung carcinomas. (1988) (35)
CD 44 v 6-targeted T cells mediate potent antitumor effects against acute myeloid leukemia and multiple myeloma (2013) (35)
Exploiting microRNA regulation for genetic engineering. (2012) (35)
Deletion in a (T)8 microsatellite abrogates expression regulation by 3'-UTR. (2003) (34)
RNAi technology and lentiviral delivery as a powerful tool to suppress Tpr-Met-mediated tumorigenesis (2005) (33)
Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens (2018) (33)
Modulation of immune responses in lentiviral vector-mediated gene transfer (2019) (32)
A double-switch vector system positively regulates transgene expression by endogenous microRNA expression (miR-ON vector). (2013) (31)
A microRNA‐Based System for Selecting and Maintaining the Pluripotent State in Human Induced Pluripotent Stem Cells (2011) (31)
Cellular innate immunity and restriction of viral infection: implications for lentiviral gene therapy in human hematopoietic cells. (2015) (30)
3 Lentiviral Vectors (1999) (29)
In Vivo Gene Delivery by Lentiviral Vectors (1999) (29)
Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome. (2013) (29)
Human glioblastoma cell lines have neuropeptide receptors for bombesin/gastrin-releasing peptide. (1989) (29)
Quantitative proteomic analysis of lentiviral vectors using 2‐DE (2009) (28)
Gene Modification and Three‐Dimensional Scaffolds as Novel Tools to Allow the Use of Postnatal Thymic Epithelial Cells for Thymus Regeneration Approaches (2019) (28)
MicroRNA-223 dose levels fine tune proliferation and differentiation in human cord blood progenitors and acute myeloid leukemia. (2015) (27)
Characterization of new arylsulfatase A gene mutations reinforces genotype‐phenotype correlation in metachromatic leukodystrophy (2009) (27)
Effects of phosphorylation and neuronal activity on the control of synapse formation by synapsin I (2011) (26)
IFNα gene/cell therapy curbs colorectal cancer colonization of the liver by acting on the hepatic microenvironment (2016) (26)
Immunological detection of proteins phosphorylated at tyrosine in cells stimulated by growth factors or transformed by retroviral-oncogene-coded tyrosine kinases. (1986) (25)
Dynamic Activity of miR-125b and miR-93 during Murine Neural Stem Cell Differentiation In Vitro and in the Subventricular Zone Neurogenic Niche (2013) (25)
Genome Editing: A Tool For Research and Therapy: Targeted genome editing hits the clinic (2014) (25)
Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next‐generation human artificial chromosomes for Duchenne muscular dystrophy (2017) (25)
A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders (2022) (24)
Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transfer. (2002) (23)
Retroviral vectors containing Tet-controlled bidirectional transcription units for simultaneous regulation of two gene activities (2006) (23)
Immune responses in liver-directed lentiviral gene therapy. (2013) (23)
Solubilization of the receptor for the neuropeptide gastrin-releasing peptide (bombesin) with functional ligand binding properties. (1990) (23)
Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts (2015) (23)
Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy (2015) (22)
Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper‐IgM syndrome (2021) (21)
Proteins phosphorylated on tyrosine as markers of human tumor cell lines (1987) (21)
developmental relationships embryonic macrophages suggests common functions and Tie 2-expressing monocytes , blood ' ' resident ' ' monocytes , and A distinguishing gene signature shared by tumor-infiltrating (2009) (21)
Manipulating Immune Tolerance with Micro-RNA Regulated Gene Therapy (2011) (19)
miR-511-3 p Modulates Genetic Programs of Tumor-Associated Macrophages (2012) (19)
Cellular and transcriptional dynamics of human neutrophils at steady state and upon stress (2022) (19)
Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies. (2016) (19)
Ectopic FOXP3 Expression Preserves Primitive Features Of Human Hematopoietic Stem Cells While Impairing Functional T Cell Differentiation (2017) (18)
Leukocytes recruited by tumor-derived HMGB1 sustain peritoneal carcinomatosis (2016) (18)
Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia (2018) (18)
Retrieval of vector integration sites from cell-free DNA (2021) (17)
MNK2 governs the macrophage antiinflammatory phenotype (2020) (17)
Short-term culture of human CD34+ cells for lentiviral gene transfer. (2009) (16)
In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors. (2002) (16)
Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease (2019) (16)
Lentiviral vector-based insertional mutagenesis identifies genes involved in the resistance to targeted anticancer therapies. (2014) (15)
Inserting optimism into gene therapy (2006) (15)
Development and maturation of invariant NKT cells in the presence of lysosomal engulfment (2009) (15)
Safer conditioning for blood stem cell transplants (2016) (15)
WFH State‐of‐the‐art paper 2020: In vivo lentiviral vector gene therapy for haemophilia (2020) (15)
Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector (2017) (15)
A receptor for bombesin with associated tyrosine kinase activity (1987) (14)
Engineered tumor-infiltrating macrophages as gene delivery vehicles for interferon-α activates immunity and inhibits breast cancer progression (2014) (14)
Targeted inducible delivery of immunoactivating cytokines reprograms glioblastoma microenvironment and inhibits growth in mouse models (2022) (13)
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy (2020) (13)
Monitoring disease evolution and treatment response in lysosomal disorders by the peripheral benzodiazepine receptor ligand PK11195 (2009) (13)
Shedding of clinical-grade lentiviral vectors is not detected in a gene therapy setting (2015) (13)
Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering (2020) (12)
Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells (2022) (11)
Distribution of nerve growth factor in chick embryo sympathetic neuronsin vitro (1981) (11)
Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency. (2021) (11)
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells (2022) (11)
Novel candidate disease for gene therapy: metachromatic leukodystrophy (2007) (10)
Detection ofphosphotyrosine-containing proteins inthedetergent- insoluble fraction ofRSV-transformed fibroblasts byazobenzene phosphonate antibodies (1984) (10)
Microtubules and microfilaments in fixed and permeabilized cells are selectively decorated by nerve growth factor. (1982) (9)
Ensuring a future for gene therapy for rare diseases (2022) (9)
Protein phosphorylation at tyrosine residues IN v‐abl transformed mouse lymphocytes and fibroblasts (1986) (9)
Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients (2020) (9)
Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy (2018) (9)
A Set of Cell Lines Derived from a Genetic Murine Glioblastoma Model Recapitulates Molecular and Morphological Characteristics of Human Tumors (2021) (8)
Good news on the clinical gene transfer front. (2008) (8)
Angiopoietin 2 expression in the cornea and its control of corneal neovascularisation (2016) (7)
Myeloid cell‐based delivery of IFN‐γ reprograms the leukemia microenvironment and induces anti‐tumoral immune responses (2021) (7)
CD44v6 Is Required For In Vivo Tumorigenesis Of Human AML and MM Cells: Role Of Microenvironmental Signals and Therapeutic Implications (2013) (7)
Visualization of phosphotyrosine containing molecules within the detergent insoluble cell matrix of v-src transformed fibroblasts. (1984) (7)
Lentiviral transduction of primary myeloma cells with CD80 and CD154 generates antimyeloma effector T cells. (2005) (7)
Title : NY-ESO-1 TCR single edited central memory and memory stem T cells to treat multiple myeloma without inducing GvHD (2017) (7)
Enriched MicroRNA-126 Bioactivity Marks the Primitive Compartment In AML and Regulates LSC Numbers (2010) (6)
Liver-Directed Gene Therapy for Hemophilia B with Immune Stealth Lentiviral Vectors (2017) (6)
In Vivo Selection for Gene-Corrected HSPCs Advances Gene Therapy for a Rare Stem Cell Disease. (2019) (6)
BAR-Seq clonal tracking of gene-edited cells (2021) (6)
Safety and Clinical Benefit of Lentiviral Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome (2015) (6)
Regulation by Hypoxia and Angiopoietin-2 Tie 2-Expressing Monocytes and Tumor Angiogenesis : (2007) (6)
Co-Expression of a Suicide Gene in CAR-Redirected T Cells Enables the Safe Targeting of CD44v6 for Leukemia and Myeloma Eradication (2012) (6)
803. Endogenous microRNA Regulation Suppresses Transgene Expression in Hematopoietic Lineages and Enables Stable Gene Transfer (2006) (6)
Therapeutic liver repopulation by transient acetaminophen selection of gene-modified hepatocytes (2021) (5)
Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010 (2019) (5)
Conditioning regimens in long term nonclinical studies to support development of ex-vivo gene therapy: review of non-proliferative and proliferative changes. (2020) (4)
481. Targeted Genome Editing in Mouse Hematopoietic Stem/Progenitor Cells (HSPC) To Model Gene Correction of SCID-X1 (2015) (4)
Intracerebral Gene Transfer Using Viral Vectors (2000) (4)
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates (2021) (4)
Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial (2019) (4)
Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Ex Vivo Gene Therapy Settings (2019) (4)
Dual Transgenesis of T Cells with a Novel CD44v6-Specific Chimeric Antigen Receptor and a Suicide Gene for Safe and Effective Targeting of Chemoresistance in Hematopoietic Tumors (2011) (3)
Efficient and sustained gene transfer in terminally differentiated cells with a lentiviral vector (1997) (3)
Characterization of the detergent solubilized receptor for gastrin-releasing peptide (1990) (3)
2. Promoter Trapping Reveals Significant Differences in Integration Site Selection Between MLV and HIV Vectors (2004) (3)
Antagonizing metastasis (2010) (3)
Identification of the Bombesin Receptor on Murine and Human Cells by Cross‐Linking Experiments (1988) (3)
Mir-126 Governs Human Leukemia Stem Cell Quiescence and Chemotherapy Resistance (2013) (2)
Abstract 2937: Editing central memory T lymphocyte specificity for safe and effective adoptive immunotherapy of leukemia (2010) (2)
The tyrosine kinase associated with the bombesin receptor complex: Evidences for autocrine activation in small cell lung carcinomas (1988) (2)
A phase I-IIa study of genetically modified Tie-2 expressing monocytes in patients with glioblastoma multiforme (TEM-GBM Study). (2021) (2)
Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy (2021) (2)
Comprehensive Clonal Mapping of Hematopoiesis in Vivo in Humans By Retroviral Vector Insertional Barcoding (2014) (2)
The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy (2022) (2)
392. Development of a New Lentiviral Vector for Site-Specific Integration into Mammalian Genomes (2005) (2)
Fighting rare diseases: the model of the telethon research institutes in Italy. (2015) (2)
TCR Gene Editing Results in Effective Immunotherapy of Leukemia without the Development of GvHD (2011) (2)
Lentiviral Vectors for Gene Delivery in the Nervous System (1998) (2)
Off-Tumor Target Expression Levels Do Not Predict CAR-T Cell Killing: A Foundation For The Safety Of CD44v6-Targeted T Cells (2013) (2)
Interferon-gamma inhibits cell replication, but not pp60src activity of RSV-transformed fibroblasts. (1987) (2)
Erratum: Preclinical Safety and Efficacy of Human CD34+ Cells Transduced with Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome (Molecular Therapy (2013) 21 (175-184) DOI:10.1038/mt.2012.23) (2016) (1)
Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs. (2022) (1)
Selective decoration of microtubules by nerve growth factor in fixed and permeabilized cells. (1983) (1)
transduction stem cells and is down-regulated by cytokines that enhance Proteasome activity restricts lentiviral gene transfer into hematopoietic (2006) (1)
TEM-GBM: An Open-Label, Phase I/IIa Dose-Escalation Study Evaluating the Safety and Efficacy of Genetically Modified Tie-2 Expressing Monocytes to Deliver IFN-α within Glioblastoma Tumor Microenvironment (2021) (1)
284MO Targeting the tumor microenvironment of glioblastoma multiforme using a macrophage-based treatment for the local delivery of immune-therapeutic payload: The TEM-GBM study (NCT03866109) (2022) (1)
42. Correction of SCID-X1 by Targeted Genome Editing of Hematopoietic Stem/Progenitor Cells (HSPC) in the Mouse Model (2016) (1)
631. Modulation of Immune Response to Gene Therapy Derived Products by CD4+ CD25+ T Regulatory Cells (2004) (1)
Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs (2022) (1)
Long-term efficacy and safety of hematopoietic stem cell gene therapy mediated by lentiviral vectors in the murine model of Wiskott-Aldrich syndrome (2008) (1)
Corrigendum to "Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models". (2009) (1)
CTIM-24. AUTOLOGOUS CD34+ ENRICHED HEMATOPOIETIC PROGENITOR CELLS GENETICALLY MODIFIED FOR HUMAN INTERFERON-α2, ARE WELL TOLERATED & RAPIDLY ENGRAFT IN PATIENTS WITH GLIOBLASTOMA MULTIFORME (TEM-GBM_001 STUDY) (2020) (1)
From TCR Gene Transfer to TCR Gene Editing of Central Memory T Lymphocytes for Immunotherapy of Leukemia. (2009) (1)
346. Gene Correction of X-Linked SCID Using Engineered Zinc Finger Nucleases and Integration Defective Lentiviral Delivery (2005) (1)
Sustained therapeutic FIX expression in liver using integration-deficient lentiviral vectors (2009) (1)
Gene therapy with hematopoietic stem and progenitor cell for monogenic disorders: a systematic review and meta-analysis (2021) (1)
Efficient transduction of CD34+CD38- cells with lentiviral vectors (1998) (1)
130. Purification of Large Scale mRNA Encoding ZFN Nucleases by dHPLC Technology (2016) (1)
Integration-defective lentiviral vectors for hemophilia: a safer platform for immune tolerance inducation and reduced genotoxicity (2010) (1)
738. Towards Gene Correction of X-Linked SCID Using Engineered Zinc Finger Nucleases and Integrase Defective Lentiviral Delivery (2006) (1)
389. Testing the Oncogenic Potential of Retroviral and Lentiviral Vector Integration (2005) (1)
Gene therapy for hemophilia A using retroviral and lentiviral vectors expressing human or canine Factor VIII (2000) (1)
Patient-specific induced pluripotent stem cells (iPSCs) as a human-derived model to study Metachromatic and Globoid Cell Leukodystrophy (2013) (1)
A Mechanistic Role For Mir-126, a Hematopoietic Stem Cell Microrna, In Acute Leukemias (2013) (1)
235. Improved Ex Vivo Gene Therapy Using Highly Purified Hematopoietic Stem and Progenitor Cells (2016) (1)
OS05.6.A Modification of the tumor microenvironment in patients with glioblastoma using autologous, genetically modified, hematopoietic stem cell-based therapy: the TEM-GBM STUDY (NCT03866109) (2022) (1)
Evidence for Efficacy and Safety of Lentiviral Mediated Gene Transfer in T Cells and CD34+ Cells from Wiskott-Aldrich Syndrome Patients. (2006) (1)
Gene-based delivery of immune-activating cytokines for cancer treatment. (2023) (1)
Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Metachromatic Leukodystrophy (MLD): Clinical Outcomes from 38 Patients (2021) (0)
Update on safety and efficacy of lentiviral hematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD) (2017) (0)
Abstract CT180: Changes in immunologic responses and in the tumor microenvironment in patients with glioblastoma multiforme treated with IFN-a immune cell and gene therapy (TEM-GBM_001 Study) (2021) (0)
Gene Therapy of Lysosomal Storage Disorders by Lentiviral Vectors (2007) (0)
Abstract SY27-02: Heterogeneity of tumor-associated macrophages: Implications for antiangiogenic therapy (2010) (0)
Evaluation of Liver Gene Therapy by Lentiviral Vectors in a Dog Model of Hemophilia B (2012) (0)
Altered regulation of receptor associated-tyrosine kinases in human tumors (1989) (0)
426. Cure of Established Disease and Direct Evidence of Cellular Cross-Correction in the Nervous System of Metachromatic Leukodystrophy Mice after HSC-Based Gene Therapy (2005) (0)
Targeted gene integration in human epidermal stem cells by Zinc-finger nuclease-mediated homologous recombination. (2012) (0)
Adopt a moratorium on heritable genome editing (2019) (0)
41. Ex Vivo Gene Therapy Provides a Unique Therapeutic Benefit and Corrects Metachromatic (MLD) and Globoid (GLD) Leukodystrophy in the Mouse Models (2004) (0)
Gene vector comprising the miRNA (2006) (0)
Long-Term Phenotypic Correction of Hemophilia A Mice Following Intravenous Injection of miRNA-Regulated Lentiviral Vectors. (2007) (0)
Deregulation of the tyrosine kinase associated with the bombesin receptor in small cell lung carcinomas (1987) (0)
330. Lentiviral Vector Mediated Hematopoietic Stem Cell Gene Therapy Combined with Non-Lethal Conditioning Restores T Cell Function in the Murine Model of Wiskott-Aldrich Syndrome (2005) (0)
408. Safety of Lysosomal Enzymes Over-Expression in HSC for Gene Therapy of Storage Disorders (2006) (0)
Ectopic FOXP3 Expression Preserves Primitive Features Of Human Hematopoietic Stem Cells While Impairing Functional T Cell Differentiation (2017) (0)
Abstract 5213: Genetically modified Tie-2 expressing monocytes target IFN-α2 to the glioblastoma tumor microenvironment (TME): Preliminary data from the TEM-GBM Phase 1/2a study (2022) (0)
P1359: USING GENE-MODIFIED AUTOLOGOUS STEM CELLS PROGENY TO REEDUCATE THE TUMOR IMMUNE MICROENVIRONMENT OF SOLID TUMORS (TEM-GBM STUDY) (2022) (0)
281. Engineering Hematopoiesis for Tumor-Targeted Interferon-alpha Delivery Inhibits Multuple Myeloma and B Cell Malignancies (2015) (0)
Overcoming the challenges in gene therapy for haemophilia (2013) (0)
972. HSC-Based Ex Vivo Gene Therapy Prolongs Survival and Improves the Phenotype of Globoid Cell Leukodystrophy Mice (2005) (0)
Abstract 3297: IFNalpha by in vivo-engineered macrophages abates liver metastases and triggers counter regulatory responses limiting efficacy (2022) (0)
The Renaissance of Gene and Cell Therapy: Florence 2016. (2016) (0)
Ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Hurler syndrome) (2021) (0)
Biffi Metachromatic Leukodystrophy Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits (2013) (0)
Identification and Function of Hematopoietic Stem and Progenitor Cell Specific Micrornas. (2010) (0)
S106: LONG-TERM FOLLOW-UP OF BETA-THALASSEMIA PATIENTS TREATED WITH HEMATOPOIETIC STEM CELL GENE THERAPY (2022) (0)
Effective liver gene therapy of hemophilia B in mice and dogs by engineering lentiviral vectors with hyper-functional transgenes (2012) (0)
36. Genome-Wide Insight Into the Transcriptional Modulations Triggered By Lentiviral Transduction in Human Hematopoietic Stem Cells (2015) (0)
Regulated lentiviral vector gene transfer for X-CGD (2012) (0)
173. Insulin B9-23 LV-Driven Expression in Hepatocytes Combined With Suboptimal Dose of Anti-CD3 mAb Cures Type 1 Diabetes in NOD Mice (2015) (0)
Liver gene therapy by lentiviral vectors provides stable clinical benefit in three hemophilia B dogs and eradicates factor IX inhibitors in mice (2013) (0)
ATIM-36. TEM-GBM-001 STUDY: AUTOLOGOUS CD34+ ENRICHED HEMATOPOIETIC PROGENITOR CELLS GENETICALLY MODIFIED FOR HUMAN INTERFERON-α2 & ADMINISTERED TO PATIENTS WITH GLIOBLASTOMA & AN UNMETHYLATED MGMT PROMOTER (2019) (0)
Abstract 3169: Lentiviral vector-based insertional mutagenesis identifies new clinically relevant liver cancer genes. (2013) (0)
Integrated use of tumor AI volumetrics and advanced MRI to assess early response to Temferon in a phase 2 GBM trial: A novel paradigm. (2021) (0)
686. Gene Correction of IL2RG in Human Hematopoietic Stem and Progenitor Cells (2015) (0)
TCR Gene Editing Achieved In a Single Round Of T Cell Activation Is Sufficient To Redirect T Cell Specificity and Prevent GvHD (2013) (0)
716. Durable Acute Myeloid Leukemia Remission Without Myeloablation in an Innovative Xenotolerant Mouse Model of CD44v6 CAR-T Cell Immunotherapy (2015) (0)
PO-040 Development of a tunable form of interferon alpha for in vivo cancer gene therapy (2018) (0)
280. Lentiviral-Mediated Gene Therapy Restores B Cell Homeostasis and Tolerance in Wiskott-Aldrich Syndrome Patients (2016) (0)
1038. Lentiviral Gene Transfer into HSC Is Enhanced by Early-Acting Cytokines without Impairing Stem Cell Properties and Involves Cellular Responses Distinct from Cell Cycle Control (2005) (0)
Evidence for efficacy and safety of lentiviral mediated gene transfer in Wiskott–Aldrich syndrome (2008) (0)
Cell-Substratum Interaction of Cultured Avian Is Mediated by Specific Adhesion Structures Osteoclasts (2003) (0)
Lentiviral haematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD): Preliminary results from a clinical trial with a cryopreserved formulation of OTL-200 (2019) (0)
Transcriptional targeting of lentiviral vectors by LTR enhancer replacement (2002) (0)
Hyper-Functional Synthetic Transgenes Improve the Efficiency of Lentiviral and AAV-Based Liver-Directed Gene Therapy and Induce Immune Tolerance (2013) (0)
Lentiviral vectors efficiently transduce human bone marrow mesenchymal cells (2003) (0)
Thymic Tissue Regeneration Using Natural Collagen Scaffolds (2014) (0)
PS1220 EX-VIVO HEMATOPOIETIC STEM CELL GENE THERAPY (GT) FOR MUCOPOLYSACCHARIDOSIS TYPE I HURLER (MPSIH): PRELIMINARY RESULTS FROM A PHASE I/II CLINICAL STUDY (2019) (0)
Abstract 2849: Tie2-expressing macrophages (TEM) depletion may enhance the clinical efficacy of combretastatin A-4-phosphate (CA-4-P) (2011) (0)
Dynamics and genomic landscape of CD8+ T cells undergoing hepatic priming (2019) (0)
TIE2-expressing monocytes regulate revascularisation of the ischaemic limb (2013) (0)
37. Towards Clinical Translation of Hematopoietic Stem Cell Gene Editing for the Correction of SCID-X1 Mutations (2016) (0)
BM-derived cells that foster tumor growth and their manipulation (2008) (0)
888. Characterization of New Murine Models of Globoid Cell Leukodystrophy: Relevance for Gene Therapy Applications and Studies on Disease Pathogenesis (2006) (0)
Targeted disruption of T cell receptor genes using talens (2014) (0)
Abstract B181: Genetic engineering of tumor-infiltrating monocytes to inhibit primary and metastatic breast cancer (2016) (0)
Activation of the met oncogene tyrosine kinase by autophosphorylation (1990) (0)
Solubilization of human bombesin/GRP receptors (1990) (0)
379TiP TEM-GBM: A phase I-IIa clinical study of genetically modified Tie-2-expressing monocytes in patients with glioblastoma multiforme (2021) (0)
Integration-defective lentiviral vectors expressing engineered hyperactive factor IX improve vector performance following hepatic transduction (2012) (0)
Towards Clinical Translation of Hematopoietic Cell Gene Editing for Treating Hyper-IgM Type 1 (2021) (0)
Adaptive Routes of Hematopoietic Stem Cell Differentiation to Disease Conditions and Age in Gene Therapy Patients (2022) (0)
981. Prevention of Transgene Expression in Antigen Presenting Cells Correlate with Modulated Immune Response after In Vivo Gene Transfer (2006) (0)
Incremental Innovation of Ex Vivo Hematopoietic Stem Cell Engineering to Expand Clinical Gene Therapy Applications (2016) (0)
Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens (2018) (0)
Macrophages, angiopoietin 2, and corneal neovascularization: translational implications (2016) (0)
IMMUNOLOGICAL DETECTION OF CELLULAR TARGETS FOR V-ONC GENE CODED (1984) (0)
Proteins phosphorylated at tyrosine residues in cells transformed by v-onc genes (1984) (0)
Hematopoietic Stem Cell Gene Therapy for Rare Diseases: Current Experience and Future Perspectives (2013) (0)
Abstract 104: New liver cancer genes identified by lentiviral vector-based insertional mutagenesis in mice are associated to differential survival in hepatocellular carcinoma patients (2012) (0)
Abstract PR13: Development of chimeric forms of IFN-alpha for “on demand” in vivo cancer gene therapy (2020) (0)
6. Targeted Genome Editing of Cell Lines for Improved and Scalable Production of Lentiviral Vectors for Human Gene Therapy (2015) (0)
Cyclosporine H Overcome s Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells Graphical (2018) (0)
1014 – CELLULAR SENESCENCE: AN UNINTENDED TWIST FOR GENE AND CELL THERAPY APPLICATIONS (2022) (0)
Lentiviral and oncoretroviral vector-based gene therapy for hemophilia A (2001) (0)
Abstract 1366: Inhibition of Tie2+ macrophage infiltration into tumors in combination with CA-4-P therapy may enhance the clinical efficacy of this vascular disrupting agent (2010) (0)
Gene transfer strategies: principles, state-of-the-art and the major barriers that need to be overcome 1 (2014) (0)
NY-ESO-1 Single Edited T Cells to Treat Multiple Myeloma without Inducing GvHD (2014) (0)
Gene correction by homologous recombination in TCIRG1-defective induced pluripotent stem cells (2015) (0)
446. Tie2 Expression Defines an Integrated System of Cell Types Specifically Involved in Angiogenesis, and Provides a Platform for Targeted Gene Delivery to Tumors (2005) (0)
286. Genome Editing of Inducible Cell Lines for Scalable Production of Improved Lentiviral Vectors for Human Gene Therapy (2016) (0)
558. Targeted Gene Therapy in CD34+ Cells from Healthy Donors and Fanconi Anemia Patients (2016) (0)
Linear Amplification Mediated PCR for the retrieval of lentiviral vector integration sites from tumors induced by insertional mutagenesis. (2013) (0)
Modeling the Genotoxicity of Viral Vector Integration in a Tumor Prone Hematopoietic Stem Cell Transplantation Model. (2006) (0)
Assessing Stealth and Sensed Base Editing in Human Hematopoietic Stem/Progenitor Cells (2021) (0)
512. The Cytokine Release Syndrome Crucially Contributes to the Anti-Leukemic Effects of CD44v6 CAR-T Cells (2016) (0)
Solubilization and Characterization of the Receptor for Gastrin-Releasing Peptide (1993) (0)
P05.02 A phase I/IIa dose escalation study evaluating the safety and efficacy of autologous CD34+ enriched hematopoietic progenitor cells genetically modified for human interferon-α2 in patients with GBM and an unmethylated MGMT promoter (TEM-GBM-001) (2019) (0)
Lentiviral and retroviral vector-based gene therapy for hemophilia A in Factor VIII-deficient mice (2001) (0)
Targeting the proangiogenic and immunosuppressive tumour microenvironment in primary and metastatic breast cancer (2016) (0)
28. Intravenous Administration of Lentiviral Vectors Expressing Hyperactive Factor IX Converts Severe Into Mild Hemophilia B in a Canine Model (2015) (0)
661. Immune System Regulation of Transgene Expression in the Brain 3: Effects of the Immune Response on Transgene Expression from HIV-Derived Lentiviral Vectors Injected into the Rat Striatum (2004) (0)
Method and medium for the production of safe lentiviral vectors recombinant high titer (2000) (0)
476. Clonal Tracking of Engineered Hematopoiesis In Vivo in Humans By Insertional Barcoding (2015) (0)
79. Systemic Administration of Lentiviral Vectors Triggers Innate Host Responses (2006) (0)
Integration site analysis by non-restrictive (nr) LAM-PCR combined with optimized high-throughput sequencing (2009) (0)
Targeting models of severe combined immunodeficiency for correction by zinc finger nucleases (2011) (0)
miRNA gene vector comprising (2006) (0)
27. Aberrant Expression of the Stem Cell microRNA-126 Induces B Cell Malignancy (2015) (0)
690. Permanent Epigenetic Silencing of Human Genes With Artificial Transcriptional Repressors (2015) (0)
Abstract 1013: Enriched miR-126 bioactivity marks the primitive compartment in human AML and regulates leukemia stem cell numbers (2012) (0)
Gene therapy for hemophilia A using onco-retroviral, lentiviral and gutless adenoviral vectors (2001) (0)
Erratum to "Lentiviral Vector-Mediated Gene Transfer in T Cells from Wiskott-Aldrich Syndrome Patients Leads to Functional Correction". (2005) (0)
Proteins phosphorylated on tyrosine as markers of human malignancies (1987) (0)
Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in nine patients treated with a cryopreserved formulation of OTL-200 (2021) (0)
In vivo tracking of human hematopoiesis reveals patterns of clonal dynamics during early post-transplant and steady-state reconstitution phases (2018) (0)
Identification and Localization of Phosphoproteins in v-onc Transformed Fibroblasts by Means of Phosphotyrosine Antibodies (1985) (0)
Lentiviral Gene Transfer is a Novel Strategy in Preventing Liver Fibrosis in LEC Rats Modeling Wilson Disease (2005) (0)
Liver Gene Therapy by Lentiviral Vectors Allows Partial Correction of Two Hemophilia B Dogs and Revert Pre-Existing Anti-Transgene Immunity in Mice (2012) (0)
906. Successful functional correction of T cells from Wiskott-Aldrich syndrome patients with lentiviral vectors (2004) (0)
891. Correction of Established Neurologic Disease and Evidences of In Vivo Cross Correction in the Mouse Model of Metachromatic Leukodystrophy (2006) (0)
therapy in hemophilic mice Hyperfunctional coagulation factor IX improves the efficacy of gene (2013) (0)
Potent In Vivo Anti-Tumor Activity Of Extracellular Vesicles Isolated From Genetically Engineered Primary Mesenchymal Stromal Cells Expressing The Trans-Membrane TNF-Related Apoptosis-Inducing Ligand (TRAIL) (2013) (0)
331. Lentivirus-Mediated Ex Vivo Gene Therapy in ADA-Deficient SCID Mice (2005) (0)
15 Correction of established neurologic disease and evidences of in vivo cross correction in the mouse model of metachromatic leukodystrophy (2007) (0)
metabolic defects deaminase (ADA)-deficient mice and corrects their immune and Ex vivo gene therapy with lentiviral vectors rescues adenosine (2012) (0)
T Cell Receptor Gene Transfer into Naive and Central Memory Lymphocytes by Lentiviral Vectors for a Safe and Effective Adoptive Immune Therapy of Leukemia (2008) (0)
High Levels of MicroRNA-126 Bioactivity Specify the LSC Compartment in AML (2008) (0)
57. Targeted Gene Delivery of Alpha-Interferon by Genetically Modified Hematopoietic Cells Inhibits Glioma Vascularization and Growth without Systemic Toxicity (2006) (0)
Preclinical safety studies for gene therapy medicinal products in GLP test facility (2014) (0)
and Efficient In Vivo Gene Delivery Self-Inactivating Lentivirus Vector for Safe (2013) (0)
864. Characterization of Transgenic Mice Over_ Expressing Arylsulfatase A: Implications for Gene Therapy and Sulfatases Activation Mechanisms (2006) (0)
University of Groningen In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases Biasco, (0)
3. Effect of Retroviral and Lentiviral Vector Integrations on Transcription of Flanking Genes (2004) (0)
1041 Gene therapy of Wilson disease with lentiviral vectors in a rat model (2003) (0)
CLRM-08 TARGETING IMMUNE-PAYLOAD TO THE GLIOBLASTOMA TUMOR MICROENVIRONMENT USING A MACROPHAGE-BASED TREATMENT RELYING ON AUTOLOGOUS, GENETICALLY MODIFIED, HEMATOPOIETIC STEM CELL-BASED THERAPY: THE TEM-GBM STUDY (NCT03866109) (2022) (0)
373. Use of Lentiviral and Adeno-Associated Vectors for Targeted Gene Inactivation in Human Cancer Cells (2005) (0)
694. Involvement of Glycosaminoglycans in VSV-G Pseudotyped Lentiviral Vector Mediated Gene Transfer into Airway Epithelial Cells (2006) (0)
Lentiviral vectors for gene therapy (1998) (0)
TEM-MM-101: A Phase I/IIa Dose Escalation Study Evaluating the Safety and Activity of Autologous CD34+ Enriched Hematopoietic Progenitor Cells Genetically Modified for Human Interferon-α2 in Multiple Myeloma Patients with Early Relapse after Intensive Front Line Therapy (2019) (0)
Lentiviral-Mediated Gene Therapy for the Treatment of Adenosine Deaminase 2 Deficiency (2021) (0)
The promise of tumor-targeted gene-based delivery of immune-activating cytokines (2019) (0)
Successful Liver Gene Therapy of Hemophilia B Dogs Using Lentiviral Vectors and Hyperfunctional Transgene (2013) (0)
Method and medium for the production of safe, recombinant lentivirus high titer (1998) (0)
209. TCR Gene Editing in a Single Step of T Cell Activation To Redirect T Cell Specificity and Prevent GvHD (2015) (0)
gene therapy transcriptional activity of T cells from ADA-SCID patients treated with Integration of retroviral vectors induces minor changes in the (2014) (0)
Lentiviral vectors: design, biological properties, milestones and current major applications, hazards 1 (2014) (0)
Abstract 4982: Identification of new human liver cancer genes by a novel lentiviral vector-based insertional mutagenesis approach in three mouse models of hepatocarcinogenesis (2011) (0)
InVivoGeneDelivery andStable Transduction of Nondividing Cells bya Lentiviral Vector (2016) (0)
36. Improved Lentiviral Vectors for Systemic Gene Transfer in the Absence of an Immune Response (2005) (0)
Selection procedure for generating efficient pack-cells for lentiviral vectors (1999) (0)
Human artificial chromosome-mediated genetic correction of human dystrophic skeletal muscle progenitors for the autologous cell therapy of Duchenne muscular dystrophy (2015) (0)
Correction of metachromatic leukodystrophies (MLD) in the mouse model by transplantation of genetically modified hematopoietic stem cells (2004) (0)
442. LV Expressing MR Reporter Genes Allows In Vivo Monitoring of Stem Cell Gene Therapy (2006) (0)
Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking (2020) (0)
Table of Contents (1984) (0)
731. Hematopoietic Stem Cell Gene Transfer and Integration Site Analysis in Tumor-Prone Mice Uncovers Low Genotoxicity of Lentiviral Vector Integration (2006) (0)
Clonal tracking of engineered human hematopoiesis through integration sites analysis (2016) (0)
in hemophilic mice Hyper-functional coagulation factor IX improves the efficacy of gene (2012) (0)
Assessing the impact of lentiviral vector integration on splicing of cellular genes at the genome-wide level (2011) (0)
Reprogramming Macrophages Using Autologous Hematopoietic Stem Cells As Immunotherapy for Glioblastoma: TEM-GBM Study (NCT03866109) (2022) (0)
Exploiting microRNA expression profiles for lineage- and differentiation state-specific transgene expression in hematopoietic, neural and embryonic stem cells (2008) (0)
First-in-human phase I/II clinical trial of hematopoietic stem and progenitor cell gene therapy for Hurler syndrome: Favorable safety profile and extensive metabolic correction (2022) (0)
Lentiviral Vector-Mediated Induction of Oct4 Expression in Primary Adult Mouse Thymic Epithelial Cells To Generate Tools for Gene Replacement and Tissue Regeneration (2014) (0)
LIVER-DIRECTED LENTIVIRAL GENE THERAPY PROVIDES STABLE BENEFIT IN HEMOPHILIA B DOGS WITHOUT EVIDENCE OF GENOTOXICITY IN SENSITIZED MOUSE MODELS (2014) (0)
Corrigendum to "Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome". (2016) (0)
3. Safety Assessment of SIN LVs Harboring Chromatin Insulators in the Sensitive Cdkn2a-/- In Vivo Genotoxicity Assay Show Enhancer-Blocking Activity of Specific Insulator Sequences (2015) (0)
Modeling the genotoxicity of viral vector integration in a tumor prone hematopoietic stem cell transplantation model (2008) (0)
Efficient generation of gene-corrected and reprogramming factor-free iPS cells from SCID-X1 patients (2012) (0)
(immunofluorescence/nerve growth factor antibodies/autoradiogi (2016) (0)
Monocyte-Macrophage Function Hepatocyte Growth Factor Is a Regulator of (2000) (0)
1003. Targeted Site-Specific Integration in Human Cells Using Designed Zinc Finger Nucleases (2006) (0)
Editing Human Lymphocyte Specificity for Safe and Effective Adoptive Immunotherapy of Leukemia. (2010) (0)
Efficient Generation of Gene-Corrected, Vector- and Reprogramming Factor-Free iPS Cells from SCID-X1 Patients (2012) (0)
332. Dual Gene Transfer and Transgenesis by Novel Lentiviral Vectors Carrying Synthetic Bi-Directional Promoters (2004) (0)
Modeling and Increasing the Genome Accessibility of Viral Integration Sites (2009) (0)
RON IS A HETERODIMERIC TYROSINE KINASE RECEPTOR ACTIVATED BY THE HGF HOMOLOG MSP / G. GAUDINO; A. FOLLENZI; L. NALDINI; C. COLLESI; M. SANTORO; (0)
345. Gene Therapy for Wiskott-Aldrich Syndrome Using Lentiviral Vectors: Evidence for Efficacy and Safety after Transduction of Human T Cells and Hematopoietic Stem Cells (2005) (0)
468. Cellular Therapy with Transgene Expressing APC Activates CD4+CD25+ Regulatory T Cells Which Modulate the Immune Response to Gene Therapy Derived Products in Immunocompetent Mice (2005) (0)
BACR/BMS Special Conference ‘‘Tumour Vasculature: New Targets & Therapies’’ 25th/28th June 2006, Cirencester, UK (2006) (0)
Receptor for bombesin with associated tyrosine kinase activity (1986) (0)
5 INVITED Role of haematopoietic cells in tumour angiogenesis: from discovery to targeted cancer gene therapy (2007) (0)
The Receptor for the Hepatocyte Growth Factor-Scatter Factor: Ligand-Dependent and Phosphorylation-Dependent Regulation of Kinase Activity (1992) (0)
65. Long-Term Effects of Hematopoietic Stem Cell Gene Therapy in the Murine Model of Wiskott-Aldrich Syndrome: Persistence of Functional Correction of T Cells and Lack of Malignant Trasformation (2006) (0)
Giulia Escobar Inhibits Breast Cancer Progression Delivery α Genetic Engineering of Hematopoiesis for Targeted IFN - (2013) (0)
Investigation on the antiproliferative activity of copper-cimetidine derivatives in neoplastic and syngeneic normal cells: role of H2-receptors (1992) (0)
Hematopoietic Stem Cell Expansion, without Exhaustion or Transformation, by Stable Microrna Antagonism in Vivo (2012) (0)
S912 LOCAL INTERFERON-ALPHA DELIVERY INTO THE MYELOMA MICROENVIRONMENT BY GENE-ENGINEERED AUTOLOGOUS STEM CELL TRANSPLANTATION (2019) (0)
Structure, Biosynthesis and Biochemical Properties of the HGF/SF Receptor (1993) (0)
OP 25 ( P 141 ) DLX 3 is a Major Regulator of Bone Apposition and Homeostasis in the Appendicular Skeleton (2015) (0)
Synthetic bi-directional promoters and their uses (2004) (0)
Su.98. Defining a Role for FOXP3 in Human Cd4+ T-Cells (2006) (0)
Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy. (2023) (0)
Immune deficiencies , infection , and systemic immune disorders B-cell reconstitution after lentiviral vector – mediated gene therapy in patients with Wiskott-Aldrich syndrome (0)
Abstracts of the 8th Meeting of the Italian Peripheral Nerve Study Group: 31: Absract (2003) (0)
756. Stable Amelioration of Hemophilia B in Dogs by Intravenous Administration of Lentiviral Vectors Expressing Hyper-Functional Factor IX (2016) (0)
ORAL PRESENTATIONS; THERAPEUTIC POTENTIAL OF GENETICALLY MODIFIED NEURAL STEM CELLS (NSCS) IN A MOUSE MODEL OF GLOBOID CELL LEUKODYSTROPHY (GLD) (2010) (0)
9. Hematopoietic stem cell based gene therapy for the treatment of metachromatic leukodystrophy: Towards clinical testing (2008) (0)
358. Gene Transfer to the Airway Epithelium Mediated by a Third Generation HIV-1 Based Vector: Efficiency and Role of Heparan Sulfate|[ast]| (2005) (0)
miR-126 Regulates Distinc t Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells Graphical (0)
Generation of Memory Stem T Cells Specific for Tumor Antigens and Resistant to Inhibitory Signals By Genome Editing (2018) (0)
103. Development of an effective and safe hematopoietic stem cell gene therapy for globoid cell leukodystrophy: The unexpected issue of GALC toxicity (2008) (0)
IMMU-01. TEM-GBM: AN OPEN-LABEL, PHASE I/IIA DOSE-ESCALATION STUDY EVALUATING THE SAFETY AND EFFICACY OF GENETICALLY MODIFIED TIE-2 EXPRESSING MONOCYTES TO DELIVER IFN-A WITHIN GLIOBLASTOMA TUMOR MICROENVIRONMENT (2021) (0)
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia (2019) (0)
295. Hematopoietic Stem Cell Gene Therapy (2.0) Based on Purified CD34+CD38- Cells (2015) (0)
752. Single Chain TCR Gene Editing in Adoptive Cell Therapy for Multiple Myeloma (2016) (0)

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