Evelyne Gicquel

Evry, 91000, France

Last Updated: 09/26/2026

Save publications for later

Sign Up

Not sure about your diagnosis?

Check Your Symptoms

Found 11 publications

Transgene-induced cardiotoxicity in high-dose AAV gene transfer.

Transgene-induced cardiotoxicity in high-dose AAV gene transfer.

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

Published: March 26, 2025

Advances and Challenges in Microdystrophin gene therapy for Duchenne Muscular Dystrophy: progress and future directions

Advances and Challenges in Microdystrophin gene therapy for Duchenne Muscular Dystrophy: progress and future directions

Journal: Medecine sciences : M/S

Published: November 18, 2024

CRISPR-Cas9 KO Cell Line Generation and Development of a Cell-Based Potency Assay for rAAV-FKRP Gene Therapy.

CRISPR-Cas9 KO Cell Line Generation and Development of a Cell-Based Potency Assay for rAAV-FKRP Gene Therapy.

Journal: Cells

Published: July 27, 2023

Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.

Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.

Journal: Journal of inherited metabolic disease

Published: December 21, 2022

Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in the dystrophic muscle in Duchenne muscular dystrophy.

Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in the dystrophic muscle in Duchenne muscular dystrophy.

Journal: Life science alliance

Published: April 28, 2022

Skeletal Muscle Cells Derived from Induced Pluripotent Stem Cells: A Platform for Limb Girdle Muscular Dystrophies.

Skeletal Muscle Cells Derived from Induced Pluripotent Stem Cells: A Platform for Limb Girdle Muscular Dystrophies.

Journal: Biomedicines

Published: April 28, 2022

Minimal Consequences of CMAH and DBA/2 Backgrounds on a FKRP Deficient Model.

Minimal Consequences of CMAH and DBA/2 Backgrounds on a FKRP Deficient Model.

Journal: Journal of neuromuscular diseases

Published: September 14, 2020

Functional and cellular localization diversity associated with Fukutin-related protein patient genetic variants.

Functional and cellular localization diversity associated with Fukutin-related protein patient genetic variants.

Journal: Human mutation

Published: November 09, 2018

AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression.

AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression.

Journal: Human molecular genetics

Published: December 07, 2016

The phenotype of dysferlin-deficient mice is not rescued by adeno-associated virus-mediated transfer of anoctamin 5.

The phenotype of dysferlin-deficient mice is not rescued by adeno-associated virus-mediated transfer of anoctamin 5.

Journal: Human gene therapy. Clinical development

Published: June 01, 2013

A human skeletal muscle interactome centered on proteins involved in muscular dystrophies: LGMD interactome.

A human skeletal muscle interactome centered on proteins involved in muscular dystrophies: LGMD interactome.

Journal: Skeletal muscle

Published: August 03, 2012

Showing 1-11 of 11

Last Updated: 09/26/2026