Gene modification strategies using AO‐mediated exon skipping and CRISPR/Cas9 as potential therapies for Duchenne muscular dystrophy patients

Duchenne muscular dystrophy (DMD) is an X‐linked genetic disease affecting 1 in 5000 young males worldwide annually. Patients experience muscle weakness and loss of ambulation at an early age, with ∼75% reduced life expectancy. Recently developed genetic editing strategies aim to convert severe DMD...

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Detalles Bibliográficos
Autores principales: Solberg, Marthe Helene, Shariatzadeh, Maryam, Wilson, Samantha L
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Institution of Engineering and Technology 2020
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9996716/
https://www.ncbi.nlm.nih.gov/pubmed/36968157
http://dx.doi.org/10.1049/enb.2020.0017

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