NICHOLSON, Michael, G.,JANTZ, Derek,SMITH, James, Jefferson
申请号：
DK15761320
公开号：
DK3116533T3
申请日:
2015.03.12
申请国别(地区):
DK
年份:
2020
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.
