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Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice

Gene therapy, 2006-09, Vol.13 (17), p.1300-1308

2006

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AAV vectors for RNA-based modulation of gene expression

Gene therapy, 2008-06, Vol.15 (11), p.864-869

Danos, O

2008

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Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients

Gene therapy, 2007-03, Vol.14 (5), p.415-428

2007

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Intracellular Trafficking of Adeno-Associated Virus Vectors: Routing to the Late Endosomal Compartment and Proteasome Degradation

Journal of Virology, 2001-02, Vol.75 (4), p.1824-1833

2001

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A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice

Gene therapy, 2005-04, Vol.12 (7), p.597-606

2005

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Lentiviral protein delivery of meganucleases in human cells mediates gene targeting and alleviates toxicity

Gene therapy, 2014-08, Vol.21 (8), p.759-766

2014

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Genetic correction of canine dystrophic epidermolysis bullosa mediated by retroviral vectors

Human molecular genetics, 2003-08, Vol.12 (15), p.1897-1905

2003

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Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

Nature genetics, 1997-07, Vol.16 (3), p.270-276

1997

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AAV-mediated delivery of a mutated myostatin propeptide ameliorates calpain 3 but not α-sarcoglycan deficiency

Gene therapy, 2007-05, Vol.14 (9), p.733-740

2007

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Safe and Efficient Generation of Recombinant Retroviruses with Amphotropic and Ecotropic Host Ranges

Proceedings of the National Academy of Sciences - PNAS, 1988-09, Vol.85 (17), p.6460-6464

1988

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Successful Interference with Cellular Immune Responses to Immunogenic Proteins Encoded by Recombinant Viral Vectors

Journal of Virology, 2001-01, Vol.75 (1), p.269-277

2001

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Linear Topology Confers in Vivo Gene Transfer Activity to Polyethylenimines

Bioconjugate chemistry, 2006-05, Vol.17 (3), p.759-765

2006

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Human immunodeficiency virus type 1 Nef increases the efficiency of reverse transcription in the infected cell

Journal of Virology, 1995-07, Vol.69 (7), p.4053-4059

1995

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HLA-A0201-restricted Cytolytic Responses to the rtTA Transactivator Dominant and Cryptic Epitopes Compromise Transgene Expression Induced by the Tetracycline on System

Molecular therapy, 2004-08, Vol.10 (2), p.279-289

2004

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Canine Adenovirus Type 2 Attachment and Internalization: Coxsackievirus-Adenovirus Receptor, Alternative Receptors, and an RGD-Independent Pathway

Journal of Virology, 2000-11, Vol.74 (22), p.10639-10649

2000

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Synthesis, Characterization, and Gene Transfer Application of Poly(ethylene glycol-b-ethylenimine) with High Molar Mass Polyamine Block

Biomacromolecules, 2006-10, Vol.7 (10), p.2863-2870

2006

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P.130 RGX-202: An investigational AAV8 gene therapy coding for a novel microdystrophin as a treatment for Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2022-10, Vol.32, p.S101

2022

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DNA compaction into new DNA vectors based on cyclodextrin polymer: Surface enhanced Raman spectroscopy characterization

Biopolymers, 2006-04, Vol.81 (5), p.360-370

2006

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Adeno-associated virus pseudotype 5 vector improves gene transfer in arthritic joints

Human gene therapy, 2005-04, Vol.16 (4), p.426-434

2005

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Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies

Gene therapy, 2006-01, Vol.13 (1), p.20-28

2006

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Efficient gene transfer in skeletal muscle with AAV-derived bicistronic vector using the FGF-1 IRES

Gene therapy, 2008-08, Vol.15 (15), p.1090-1098

2008

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Gene Therapy Protocols

British Journal of Cancer, 2009-03, Vol.100 (6), p.1018-1019

Danos, O

2009

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Myogenic program induction in mature fat tissue (with MyoD expression)

Experimental cell research, 2005-08, Vol.308 (2), p.300-308

2005

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Rescue of Dystrophic Muscle through U7 snRNA-Mediated Exon Skipping

Science (American Association for the Advancement of Science), 2004-12, Vol.306 (5702), p.1796-1799

2004

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P16 An investigational AAV8 gene therapy coding for a novel microdystrophin as a treatment for Duchenne muscular dystrophy

Neuromuscular disorders : NMD, 2023-10, Vol.33, p.S101-S101

2023

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Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice

AAV vectors for RNA-based modulation of gene expression

Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients

Intracellular Trafficking of Adeno-Associated Virus Vectors: Routing to the Late Endosomal Compartment and Proteasome Degradation

A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice

Lentiviral protein delivery of meganucleases in human cells mediates gene targeting and alleviates toxicity

Genetic correction of canine dystrophic epidermolysis bullosa mediated by retroviral vectors

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

AAV-mediated delivery of a mutated myostatin propeptide ameliorates calpain 3 but not α-sarcoglycan deficiency

Safe and Efficient Generation of Recombinant Retroviruses with Amphotropic and Ecotropic Host Ranges

Successful Interference with Cellular Immune Responses to Immunogenic Proteins Encoded by Recombinant Viral Vectors

Linear Topology Confers in Vivo Gene Transfer Activity to Polyethylenimines

Human immunodeficiency virus type 1 Nef increases the efficiency of reverse transcription in the infected cell

HLA-A0201-restricted Cytolytic Responses to the rtTA Transactivator Dominant and Cryptic Epitopes Compromise Transgene Expression Induced by the Tetracycline on System

Canine Adenovirus Type 2 Attachment and Internalization: Coxsackievirus-Adenovirus Receptor, Alternative Receptors, and an RGD-Independent Pathway

Synthesis, Characterization, and Gene Transfer Application of Poly(ethylene glycol-b-ethylenimine) with High Molar Mass Polyamine Block

P.130 RGX-202: An investigational AAV8 gene therapy coding for a novel microdystrophin as a treatment for Duchenne muscular dystrophy

DNA compaction into new DNA vectors based on cyclodextrin polymer: Surface enhanced Raman spectroscopy characterization

Adeno-associated virus pseudotype 5 vector improves gene transfer in arthritic joints

Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies

Efficient gene transfer in skeletal muscle with AAV-derived bicistronic vector using the FGF-1 IRES

Gene Therapy Protocols

Myogenic program induction in mature fat tissue (with MyoD expression)

Rescue of Dystrophic Muscle through U7 snRNA-Mediated Exon Skipping

P16 An investigational AAV8 gene therapy coding for a novel microdystrophin as a treatment for Duchenne muscular dystrophy

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