Jacques-P. Tremblay

Molecular characterization of a new R1925X point mutation mouse model for dysferlinopathypublié le 2025-10

In Vitro Correction of Point Mutations in the DYSF Gene Using Prime Editingpublié le 2025-06-12

Universal Prime Editing Therapeutic Strategy for RyR1-Related Myopathies: A Protective Mutation Rescues Leaky RyR1 Channelpublié le 2025-03

Emerging Perspectives on Prime Editor Delivery to the Brainpublié le 2024-06-11

Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathypublié le 2023-12-22

CRISPR/Cas9 delivery strategies with engineered extracellular vesiclespublié le 2023-09

Portrait of Dysferlinopathy: Diagnosis and Development of Therapypublié le 2023-09-16

Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9publié le 2023-08

Finding an Appropriate Mouse Model to Study the Impact of a Treatment for Friedreich Ataxia on the Behavioral Phenotypepublié le 2023-08-19

Limb–Girdle Muscular Dystrophies Classification and Therapiespublié le 2023-07-19

Prime Editing for Human Gene Therapy: Where Are We Now?publié le 2023-02-07

Small-molecule inhibitors of proteasome increase CjCas9 protein stabilitypublié le 2023-01-19

CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editingpublié le 2022

Insertion of the Icelandic Mutation (A673T) by Prime Editing: A Potential Preventive Treatment for Familial and Sporadic Alzheimer's Diseasepublié le 2022

Serum Extracellular Vesicles for Delivery of Crispr-Cas9 Ribonucleoproteins to Modify the Dystrophin Genepublié le 2022

Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophypublié le 2022

CRISPR-Cas9 Gene Therapy for Duchenne Muscular Dystrophypublié le 2022

Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD genepublié le 2022-12

Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapypublié le 2022-10-07

Three Decades of Clinical Gene Therapy: From Experimental Technologies to Viable Treatmentspublié le 2021

Base editing strategy for insertion of the A673T mutation in the APP gene to prevent the development of AD in vitropublié le 2021

Current Clinical Applications of In Vivo Gene Therapy with AAVspublié le 2021

Myogenic progenitor cells derived from human induced pluripotent stem cell are immune-tolerated in humanized micepublié le 2021