#SeminarIRSJD · Development of gene therapies for neuromuscular diseases
- Dra. Ana Maria Buj-Bello, investigadora convidada de l'INSERM (França).
Seminari exclusiu per personal de la institució.
Gene transfer using recombinant adeno-associated viral (AAV) vectors represents a promising approach to treat human diseases. In this presentation, I will provide an overview of our work on the development of AAV-based gene therapies for two severe pediatric diseases, X-linked myotubular myopathy (XLMTM) and spinal muscular atrophy with progressive myoclonic epilepsy (SMA-PME), and current challenges in the field.