Bernard Gjata

Evry, 91000, France

Last Updated: 09/26/2026

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Found 16 publications

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.

Journal: Nature communications

Published: February 16, 2021

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.

Journal: EBioMedicine

Published: June 15, 2020

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: November 13, 2019

Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer.

Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer.

Journal: Molecular therapy. Methods & clinical development

Published: June 20, 2019

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.

Journal: Molecular therapy. Methods & clinical development

Published: May 17, 2019

An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress.

An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress.

Journal: Molecular therapy. Methods & clinical development

Published: October 17, 2018

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice.

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice.

Journal: Molecular therapy. Methods & clinical development

Published: September 11, 2018

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.

Journal: Molecular therapy. Methods & clinical development

Published: October 21, 2017

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: June 28, 2017

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Journal: Science translational medicine

Published: December 23, 2016

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

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

Journal: Nature communications

Published: August 18, 2016

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

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

Journal: Molecular therapy. Methods & clinical development

Published: May 22, 2016

Showing 1-12 of 16

Last Updated: 09/26/2026