Publication – Page 3 – Généthon

Generation of two iPSC lines from patients with inherited central core disease and concurrent malignant hyperthermia caused by dominant missense variants in the RYR1 gene

August 19, 2024

The myotubular and centronuclear myopathy patient registry: a multifunctional tool for translational research

August 19, 2024

Pharmacometabolomics applied to low-dose interleukin-2 treatment in amyotrophic lateral sclerosis

August 19, 2024

Basic notions about gene therapy from the nucleic acid perspective and applications in a pediatric disease: Duchenne muscular dystrophy.

December 18, 2023

Lessons learned from clinical experience with AAV vectors.

December 18, 2023

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome

December 11, 2023

Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial

December 11, 2023

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

December 11, 2023

Pathological modeling of glycogen storage disease type III with CRISPR/Cas9 edited human pluripotent stem cells

December 11, 2023

Generation of three induced pluripotent stem cell lines from patients with glycogen storage disease type III

December 11, 2023