Reading through premature stop codons with PTC1 24. Project Catalyst to find more Duchenne drugs. Interview by Guenter Scheuerbrandt.
作者信息
Welch Ellen, Goetz Diane, Almstead Neil
出版信息
Acta Myol. 2008 Oct;27:63-8.
PMID:19364064
Abstract
摘要
相似文献
1
2
[Duchenne muscular dystrophy].
MMW Fortschr Med. 2015 Jul 23;157(13):81. doi: 10.1007/s15006-015-3369-7.
3
Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).
J Child Neurol. 2010 Sep;25(9):1158-64. doi: 10.1177/0883073810371129. Epub 2010 Jun 2.
4
Exon skipping, a therapy for Duchenne muscular dystrophy. Interview by Guenter Scheuerbrandt.
Acta Myol. 2008 Oct;27:69-73.
5
The functional mechanisms and clinical application of read-through drugs.
Yi Chuan. 2016 Jul 20;38(7):623-633. doi: 10.16288/j.yczz.15-519.
6
Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis.
Am J Respir Crit Care Med. 2010 Nov 15;182(10):1262-72. doi: 10.1164/rccm.201001-0137OC. Epub 2010 Jul 9.
7
8
PTC124, nonsense mutations and Duchenne muscular dystrophy.
Neuromuscul Disord. 2007 Oct;17(9-10):719-20. doi: 10.1016/j.nmd.2007.07.001.
9
European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene.
Neuromuscul Disord. 2015 Jan;25(1):5-13. doi: 10.1016/j.nmd.2014.11.011. Epub 2014 Nov 24.
10
Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.
Lancet. 2008 Aug 30;372(9640):719-27. doi: 10.1016/S0140-6736(08)61168-X. Epub 2008 Aug 20.
引用本文的文献
1
Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents.
Appl Clin Genet. 2011 Mar 10;4:29-44. doi: 10.2147/TACG.S8762. Print 2011.
Zotero 插件