Enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain.

ABSTRACT: Gene replacement therapy is becoming a therapeutic option for patients with Duchenne muscular dystrophy. Truncated dystrophins that can be expressed from adeno-associated viral (AAV) vectors have been designed and shown to retain many, though not all, functional features of the full-length protein. These "microdystrophins" differ by their combination of hinges and spectrin-like repeats and the inclusion of functional domains. Several microdystrophins have advanced to the clinic, and although all have been shown to restore muscle force in preclinical models, they may differ in the efficiency of providing muscle resilience and resistance to contraction-induced stress. Here, we examine whether the inclusion of a highly evolutionarily conserved domain found at the C-Terminus of dystrophin may

SUBMITTER: Foltz SJ 

PROVIDER: S-EPMC12537463 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature