JACQUES P. TREMBLAY,JEAN-PAUL IYOMBE-ENGEMBE,PIERRE CHAPDELAINE
申请号：
US15762316
公开号：
US20180265859A1
申请日:
2016.09.23
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Methods of modifying a dystrophin gene are disclosed, for restoring dystrophin expression within a cell having an endogenous frameshift mutation within the dystrophin gene. The methods comprising introducing a first cut within an exon of the dystrophin gene creating a first exon end, wherein said first cut is located upstream of the endogenous frameshift mutation； and introducing a second cut within an exon of the dystrophin gene creating a second exon end, wherein said second cut is located downstream of the frameshift mutation. Upon joining/ligation of said first and second exon ends dystrophin expression is restored, as the correct reading frame is restored. Reagents and uses of the method are also disclosed, for example to treat a subject suffering from muscular dystrophy.
