Derek JANTZ,James Jefferson SMITH,Michael G. NICHOLSON
申请号：
US15124984
公开号：
US20170106055A1
申请日:
2015.03.12
申请国别(地区):
US
年份:
2017
代理人:
摘要：
The invention relates to the field of molecular biology and recombinant nucleic acid technology. In particular, the invention relates to methods of treating patients with Duchenne Muscular Dystrophy comprising the removal of at least one exon from the dystrophin gene using engineered nucleases to restore the normal reading frame. Further disclosed are engineered nucleases suitable for using the methods.
