RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL;UNIVERSITY OF WESTERN AUSTRALIA;RESEARCH INSTITUTE AT NATIONWIDE CHILDRENS HOSPITAL
发明人:
FLANIGAN, KEVIN,WEIN, NICOLAS,WILTON, STEPHEN,WILTON, STEVEN
申请号：
CA2957661
公开号：
CA2957661A1
申请日:
2015.08.07
申请国别(地区):
CA
年份:
2016
代理人:
摘要：
The present invention relates to the delivery of oligomers for treating patients with a 5' mutation in their DMD gene other than a DMD exon 2 duplication. The invention provides methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene resulting in translation of a functional truncated isoform of dystrophin. The methods and materials can be used for the treatment of muscular dystrophies arising from 5' mutations in the DMD gene such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
