Nicolas Guerchet

1bis, Rue De L'Internationale
Evry, 91000, France

Last Updated: 09/26/2026

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Found 10 publications

Death following high-dose AAV9 gene therapy in a patient with advanced SMA-PME.

Death following high-dose AAV9 gene therapy in a patient with advanced SMA-PME.

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

Published: November 21, 2025

Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.

Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.

Journal: Molecular therapy. Methods & clinical development

Published: April 26, 2024

An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across species.

An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across species.

Journal: Nature communications

Published: October 25, 2023

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders.

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders.

Journal: Science advances

Published: September 21, 2022

Co-Administration of Simvastatin Does Not Potentiate the Benefit of Gene Therapy in the mdx Mouse Model for Duchenne Muscular Dystrophy.

Co-Administration of Simvastatin Does Not Potentiate the Benefit of Gene Therapy in the mdx Mouse Model for Duchenne Muscular Dystrophy.

Journal: International journal of molecular sciences

Published: December 08, 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

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

Cardiac Characterization of sgca-Null Mice Using High Resolution Echocardiography.

Cardiac Characterization of sgca-Null Mice Using High Resolution Echocardiography.

Journal: Neurology international

Published: July 26, 2013

Cardiac characterization of mdx mice using high-resolution doppler echocardiography.

Cardiac characterization of mdx mice using high-resolution doppler echocardiography.

Journal: Journal of ultrasound in medicine : official journal of the American Institute of Ultrasound in Medicine

Published: April 27, 2013

Showing 1-10 of 10

Last Updated: 09/26/2026