Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors

Duchenne muscular dystrophy is an X-linked monogenic disease caused by mutations in the dystrophin gene (DMD) characterized by progressive muscle weakness, leading to loss of ambulation and decreased life expectancy. Since the current standard of care for Duchenne muscular dystrophy is to merely tre...

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Detalles Bibliográficos
Autores principales: Gapinske, Michael, Winter, Jackson, Swami, Devyani, Gapinske, Lauren, Woods, Wendy S., Shirguppe, Shraddha, Miskalis, Angelo, Busza, Anna, Joulani, Dana, Kao, Collin J., Kostan, Kurt, Bigot, Anne, Bashir, Rashid, Perez-Pinera, Pablo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10448430/
https://www.ncbi.nlm.nih.gov/pubmed/37637209
http://dx.doi.org/10.1016/j.omtn.2023.07.029

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10448430/
https://www.ncbi.nlm.nih.gov/pubmed/37637209
http://dx.doi.org/10.1016/j.omtn.2023.07.029

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