Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice

Antisense oligonucleotide (AO)-mediated exon-skipping therapeutics shows great promise for Duchenne muscular dystrophy (DMD) patients. However, recent failure with drisapersen, an AO candidate drug in phase 3 trial, highlights the importance of exploring other effective AO chemistries for DMD. Previ...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no:Mol Ther Nucleic Acids
Main Authors: Gao, Xianjun, Shen, Xiaoyong, Dong, Xue, Ran, Ning, Han, Gang, Cao, Limin, Gu, Ben, Yin, HaiFang
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2015
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4881755/
https://ncbi.nlm.nih.gov/pubmed/26440599
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2015.27
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados