RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL;UNIVERSITY OF WESTERN AUSTRALIA
发明人:
Kevin Flanigan,Nicolas Wein,Steven Wilton
申请号：
US15502702
公开号：
US20170218366A1
申请日:
2015.08.07
申请国别(地区):
US
年份:
2017
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.
