Wilton, Stephen Donald,Fletcher, Sue,McClorey, Graham
申请号：
HUE10004274
公开号：
HUE028632T2
申请日:
2005.06.28
申请国别(地区):
HU
年份:
2016
代理人:
摘要：
The invention relates to antisense oligonucleotides hybridizable to target regions in exons 53, 33, 45 or 50 of the Dystrophin gene, which oligonucleotides may comprise a non-natural backbone containing non-natural sugar moieties and/or non-natural inter-nucleotide linkages, and the use of such oligonucleotides in the treatment of muscular dystrophy, e.g. Duchenne Muscular Dystrophy.
