Therapeutic exon skipping for dysferlinopathies?

Antisense-mediated exon skipping is a promising therapeutic approach for Duchenne muscular dystrophy (DMD) currently tested in clinical trials. The aim is to reframe dystrophin transcripts using antisense oligonucleotides (AONs). These hide an exon from the splicing machinery to induce exon skipping...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Main Authors: Aartsma-Rus, Annemieke, Singh, Kavita H K, Fokkema, Ivo F A C, Ginjaar, Ieke B, van Ommen, Gert-Jan, Dunnen, Johan T den, van der Maarel, Silvère M
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2010
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2987387/
https://ncbi.nlm.nih.gov/pubmed/20145676
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ejhg.2010.4
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!

Lignende værker