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Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Nature communications, 2017-07, Vol.8 (1), p.16105-15, Article 16105

2017

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Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System

Molecular therapy. Nucleic acids, 2017-06, Vol.7 (C), p.11-19

2017

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Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

Molecular therapy. Methods & clinical development, 2019-03, Vol.12, p.157-174

2019

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VCN Analysis Using Droplet Digital PCR Method in Hematopoietic Stem Cells and T Lymphocytes after Lentiviral Transduction: Optimization and Limitations

Re:GEN open, 2022-01, Vol.2 (1), p.28-36

2022

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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16049-16049, Article 16049

2016

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Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease

Molecular therapy. Methods & clinical development, 2018-12, Vol.11, p.167-179

2018

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Vectofusin-1, a New Viral Entry Enhancer, Strongly Promotes Lentiviral Transduction of Human Hematopoietic Stem Cells

Molecular therapy. Nucleic acids, 2013, Vol.2 (5), p.e90-e90, Article e90

2013

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Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16060-16060, Article 16060

2016

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An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

Molecular therapy. Methods & clinical development, 2018-09, Vol.10, p.268-280

2018

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A Conserved Mechanism of Retrovirus Restriction in Mammals

Proceedings of the National Academy of Sciences - PNAS, 2000-10, Vol.97 (22), p.12295-12299

2000

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Safety and Efficacy of AAV-Mediated Calpain 3 Gene Transfer in a Mouse Model of Limb-Girdle Muscular Dystrophy Type 2A

Molecular therapy, 2006-02, Vol.13 (2), p.250-259

2006

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Highly efficient transduction of human plasmacytoid dendritic cells without phenotypic and functional maturation

Journal of translational medicine, 2009-01, Vol.7 (1), p.10-10, Article 10

2009

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Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy

Science translational medicine, 2014-01, Vol.6 (220), p.220ra10

2014

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Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors

The Journal of immunology (1950), 2012-06, Vol.188 (12), p.6418-6424

2012

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Liver-directed lentiviral gene therapy in a dog model of hemophilia B

Science translational medicine, 2015-03, Vol.7 (277), p.277ra28

2015

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131. Targeted Genome Editing in Spinal Muscular Atrophy

Molecular therapy, 2015-05, Vol.23, p.S53-S54

2015

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Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice

Journal of neuropathology and experimental neurology, 2018-04, Vol.77 (4), p.282-295

2018

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322. Genome Editing for Nucleotide Repeat Disorders: Towards a New Therapeutic Approach for Myotonic Dystrophy Type 1

Molecular therapy, 2016-05, Vol.24, p.S129-S130

2016

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Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice

Journal of neuropathology and experimental neurology, 2018-04, Vol.77 (4), p.282-295

2018

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Infectivity enhancement of different HIV-1-based lentiviral pseudotypes in presence of the cationic amphipathic peptide LAH4-L1

Journal of virological methods, 2013-05, Vol.189 (2), p.375-378

2013

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576. A Novel Gene Editing-Based Strategy for Myotonic Dystrophy Type 1

Molecular therapy, 2015-05, Vol.23, p.S229-S229

2015

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759. Reduction of HLH-like Manifestations in Murine Model of Munc13-4 Deficiency Following Lentiviral Gene Transfer into Hematopoietic Stem and Progenitor Cells

Molecular therapy, 2016-05, Vol.24, p.S300-S300

2016

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Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primates

Translational research : the journal of laboratory and clinical medicine, 2017-10, Vol.188, p.40-57.e4

2017

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560. Generation of Functional Regulatory T Cells by FOXP3 Gene Transfer into CD4 T Cell from IPEX Patients

Molecular therapy, 2016-05, Vol.24, p.S224-S224

2016

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690. Development of a Clinical Lentiviral Vector for Gene Therapy of SCID-X1

Molecular therapy, 2016-05, Vol.24, p.S273-S274

2016

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Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

VCN Analysis Using Droplet Digital PCR Method in Hematopoietic Stem Cells and T Lymphocytes after Lentiviral Transduction: Optimization and Limitations

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease

Vectofusin-1, a New Viral Entry Enhancer, Strongly Promotes Lentiviral Transduction of Human Hematopoietic Stem Cells

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

A Conserved Mechanism of Retrovirus Restriction in Mammals

Safety and Efficacy of AAV-Mediated Calpain 3 Gene Transfer in a Mouse Model of Limb-Girdle Muscular Dystrophy Type 2A

Highly efficient transduction of human plasmacytoid dendritic cells without phenotypic and functional maturation

Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy

Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

131. Targeted Genome Editing in Spinal Muscular Atrophy

Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice

322. Genome Editing for Nucleotide Repeat Disorders: Towards a New Therapeutic Approach for Myotonic Dystrophy Type 1

Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice

Infectivity enhancement of different HIV-1-based lentiviral pseudotypes in presence of the cationic amphipathic peptide LAH4-L1

576. A Novel Gene Editing-Based Strategy for Myotonic Dystrophy Type 1

759. Reduction of HLH-like Manifestations in Murine Model of Munc13-4 Deficiency Following Lentiviral Gene Transfer into Hematopoietic Stem and Progenitor Cells

Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primates

560. Generation of Functional Regulatory T Cells by FOXP3 Gene Transfer into CD4 T Cell from IPEX Patients

690. Development of a Clinical Lentiviral Vector for Gene Therapy of SCID-X1

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