Systemic MyoAAV-saRNA delivery activates endogenous utrophin and rescues dystrophic pathology in mdx mice

Background: Therapeutic activation of endogenous utrophin, a homolog of dystrophin, represents a mutation-agnostic therapeutic strategy with considerable potential for treating Duchenne muscular dystrophy (DMD). However, the large size of current dCas9-based activation systems hinders efficient aden...

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Detalhes bibliográficos

Principais autores:	Zhikai Xiahou, Tianyuan Wang, Zilian Zhang, Na Li, Hong Xiao, Zhuo Li, Yanming Gan, Xinwei Hou, Yafeng Song
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2025-12-01
coleção:	Regenerative Therapy
Assuntos:	Duchenne muscular dystrophy Gene therapy MyoAAV saRNA Utrophin
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2352320425002081
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