The Regents of the University of Colorado, a body corporate;Murdoch University
发明人:
Marvin Caruthers,Sibasish Paul,Rakesh N. Veedu,Katarzyna Jastrzebska,Heera Krishna
申请号：
US16825918
公开号：
US20200308586A1
申请日:
2020.03.20
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Improved compositions and methods for treating a disease or disorder through target exon skipping, and preferably muscular dystrophy by administering antisense thiomorpholino molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping to produce a functional Dystrophin protein.
