Jantz, Derek,Smith, James Jefferson,Nicholson, Michael G.
申请号:
AU2015229299
公开号:
AU2015229299A1
申请日:
2015.03.12
申请国别(地区):
AU
年份:
2016
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.