Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence

Duchenne muscular dystrophy (DMD) is an X-linked recessive, infancy-onset neuromuscular disorder characterized by progressive muscle weakness and atrophy, leading to delay of motor milestones, loss of autonomous ambulation, respiratory failure, cardiomyopathy, and premature death. DMD originates fro...

Descripción completa

Detalles Bibliográficos
Autores principales: Manini, Arianna, Abati, Elena, Nuredini, Andi, Corti, Stefania, Comi, Giacomo Pietro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Neurology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8797140/
https://www.ncbi.nlm.nih.gov/pubmed/35095747
http://dx.doi.org/10.3389/fneur.2021.814174

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8797140/
https://www.ncbi.nlm.nih.gov/pubmed/35095747
http://dx.doi.org/10.3389/fneur.2021.814174

Ejemplares similares