Antisense Oligonucleotide-Mediated Exon-skipping Therapies: Precision Medicine Spreading from Duchenne Muscular Dystrophy

In 1995, we were the first to propose antisense oligonucleotide (ASO)-mediated exon-skipping therapy for the treatment of Duchenne muscular dystrophy (DMD), a noncurable, progressive muscle-wasting disease. DMD is caused by deletion mutations in one or more exons of the DMD gene that shift the trans...

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Detalles Bibliográficos
Autor principal: Matsuo, Masafumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Japan Medical Association 2021
Materias:
Review Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8355726/
https://www.ncbi.nlm.nih.gov/pubmed/34414317
http://dx.doi.org/10.31662/jmaj.2021-0019

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8355726/
https://www.ncbi.nlm.nih.gov/pubmed/34414317
http://dx.doi.org/10.31662/jmaj.2021-0019

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