Full-length human dystrophin on human artificial chromosome compensates for mouse dystrophin deficiency in a Duchenne muscular dystrophy mouse model

Dystrophin maintains membrane integrity as a sarcolemmal protein. Dystrophin mutations lead to Duchenne muscular dystrophy, an X-linked recessive disorder. Since dystrophin is one of the largest genes consisting of 79 exons in the human genome, delivering a full-length dystrophin using virus vectors...

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Detalles Bibliográficos
Autores principales: Hiramuki, Yosuke, Abe, Satoshi, Uno, Narumi, Kazuki, Kanako, Takata, Shuta, Miyamoto, Hitomaru, Takayama, Haruka, Morimoto, Kayoko, Takehara, Shoko, Osaki, Mitsuhiko, Tanihata, Jun, Takeda, Shin’ichi, Tomizuka, Kazuma, Oshimura, Mitsuo, Kazuki, Yasuhiro
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10020543/
https://www.ncbi.nlm.nih.gov/pubmed/36928364
http://dx.doi.org/10.1038/s41598-023-31481-3

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