Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy

Dysferlinopathy is a progressive myopathy caused by mutations in the dysferlin (DYSF) gene. Dysferlin protein plays a major role in plasma-membrane resealing. Some patients with DYSF deletion mutations exhibit mild symptoms, suggesting some regions of DYSF can be removed without significantly impact...

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Detalles Bibliográficos
Autores principales: Lee, Joshua J.A., Maruyama, Rika, Duddy, William, Sakurai, Hidetoshi, Yokota, Toshifumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6234522/
https://www.ncbi.nlm.nih.gov/pubmed/30439648
http://dx.doi.org/10.1016/j.omtn.2018.10.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6234522/
https://www.ncbi.nlm.nih.gov/pubmed/30439648
http://dx.doi.org/10.1016/j.omtn.2018.10.004

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