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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2023
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Materias: | |
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 |
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