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

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...

Full description

Bibliographic Details
Main Authors: Flavien Bizot, Remko Goossens, Thomas Tensorer, Sergei Dmitriev, Luis Garcia, Annemieke Aartsma-Rus, Pietro Spitali, Aurélie Goyenvalle
Format: Article
Language:English
Published: Elsevier 2022-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: antisense oligonucleotides
exon skipping
RNA
transcript imbalance
histone deacetylase inhibitors
Duchenne muscular dystrophy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122003092

Internet

http://www.sciencedirect.com/science/article/pii/S2162253122003092

Similar Items