Renadirsen, a Novel 2′OMeRNA/ENA(®) Chimera Antisense Oligonucleotide, Induces Robust Exon 45 Skipping for Dystrophin In Vivo

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disease caused by out-of-frame or nonsense mutation in the dystrophin gene. It begins with a loss of ambulation between 9 and 14 years of age, followed by various other symptoms including cardiac dysfunction. Exon skipping of patients...

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Detalles Bibliográficos
Autores principales: Ito, Kentaro, Takakusa, Hideo, Kakuta, Masayo, Kanda, Akira, Takagi, Nana, Nagase, Hiroyuki, Watanabe, Nobuaki, Asano, Daigo, Goda, Ryoya, Masuda, Takeshi, Nakamura, Akifumi, Onishi, Yoshiyuki, Onoda, Toshio, Koizumi, Makoto, Takeshima, Yasuhiro, Matsuo, Masafumi, Takaishi, Kiyosumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8928966/
https://www.ncbi.nlm.nih.gov/pubmed/34698059
http://dx.doi.org/10.3390/cimb43030090

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