Jacques-P. Tremblay

Prime Editing Mediated Generation and Correction of the mdx5cv Mutation Restores Dystrophin Expression in Myoblastspublié le 2026-08-01

Molecular characterization of a new R1925X point mutation mouse model for dysferlinopathypublié le 2026-05

Gene Editing Strategies for Duchenne Muscular Dystrophy: From Molecular Mechanisms to Clinical Translationpublié le 2026-05-07

Successful In Vitro Modification of the Dmd Gene Using Prime Editingpublié le 2026-04-22

The Improvements and Applications of Prime Editingpublié le 2026-03-20

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

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

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