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Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse

Duchenne muscular dystrophy (DMD) is a severe muscle wasting disease arising from defects in the dystrophin gene, typically nonsense or frameshift mutations, that preclude the synthesis of a functional protein. A milder, allelic version of the disease, Becker muscular dystrophy, generally arises fro...

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Publicado no:Proc Natl Acad Sci U S A
Principais autores: Mann, Christopher J., Honeyman, Kaite, Cheng, Andy J., Ly, Tina, Lloyd, Frances, Fletcher, Sue, Morgan, Jennifer E., Partridge, Terry A., Wilton, Stephen D.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2000
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC14541/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/11120883/
https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.011408598
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