Silvère Maria Van Der Maarel,Garrit-Jan B. Van Ommen,Annemieke Aartsma-Rus,Isabella Houweling-Gazzoli,Johannes T. Den Dunnen,Van Der, Maarel Silvère Maria,Van Ommen, Garrit-jan B.,Aartsma-rus, Annemieke,Houweling-gazzoli, Isabella,Den Dunnen, Johannes T.
申请号：
US13504907
公开号：
US20120270930A1
申请日:
2009.10.29
申请国别(地区):
US
年份:
2012
代理人:
摘要：
The disclosure provides methods and compositions for inducing exon-skipping in a dysferlin pre-mRNA useful, e.g., in restoring function in a dysferlin deficiency. The disclosure also provides improved methods and compositions for generally inducing exon-skipping in a pre-mRNA.
