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CRISPR-Based Therapeutic Gene Editing for Duchenne Muscular Dystrophy: Advances, Challenges and Perspectives

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease arising from loss-of-function mutations in the dystrophin gene and characterized by progressive muscle degeneration, respiratory insufficiency, cardiac failure, and premature death by the age of thirty. Albeit DMD is one of the most...

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Detalles Bibliográficos
Autores principales: Chen, Guofang, Wei, Tingyi, Yang, Hui, Li, Guoling, Li, Haisen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9564082/
https://www.ncbi.nlm.nih.gov/pubmed/36230926
http://dx.doi.org/10.3390/cells11192964