The Regents of The University of Colorado, a body corporate;Murdoch University;Heera Krishna;Katarzyna Jastrzebska
发明人:
KRISHNA, Heera,JASTRZEBSKA, Katarzyna,CARUTHERS, Marvin,PAUL, Sibasish,VEEDU, Rakesh N.
申请号：
AU2018337854
公开号：
AU2018337854A1
申请日:
2018.09.20
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
Improved compositions and methods for treating 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.
