Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice

Antisense-mediated exon skipping is one of the most promising therapeutic strategies for Duchenne muscular dystrophy (DMD), and some antisense oligonucleotide (ASO) drugs have already been approved by the US FDA despite their low efficacy. The potential of this therapy is still limited by several ch...

Descripción completa

Detalles Bibliográficos
Autores principales: Bizot, Flavien, Goossens, Remko, Tensorer, Thomas, Dmitriev, Sergei, Garcia, Luis, Aartsma-Rus, Annemieke, Spitali, Pietro, Goyenvalle, Aurélie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9722397/
https://www.ncbi.nlm.nih.gov/pubmed/36514350
http://dx.doi.org/10.1016/j.omtn.2022.11.017

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9722397/
https://www.ncbi.nlm.nih.gov/pubmed/36514350
http://dx.doi.org/10.1016/j.omtn.2022.11.017

Ejemplares similares