Strategies for Bottlenecks of rAAV-Mediated Expression in Skeletal and Cardiac Muscle of Duchenne Muscular Dystrophy

Strategies for Bottlenecks of rAAV-Mediated Expression in Skeletal and Cardiac Muscle of Duchenne Muscular Dystrophy

Gene therapy using the adeno-associated virus (rAAV) to deliver mini/micro- dystrophin is the current promising strategy for Duchenne Muscular Dystrophy (DMD). However, the further transformation of this strategy still faces many “bottlenecks”. Most gene therapies are only suitable for infants with...

Full description

Bibliographic Details
Main Authors: Na Li, Yafeng Song
Format: Article
Language:English
Published: MDPI AG 2022-11-01
Series:Genes
Subjects:
Duchenne muscular dystrophy
gene therapy
MyoAAV
CDCs
saRNA
5′UTR
Online Access:https://www.mdpi.com/2073-4425/13/11/2021

Similar Items

Similar Items