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Compositions for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy and methods of use
专利权人:
The State University of New Jersey;Rutgers
发明人:
Babu, Gopal J.
申请号:
AU2018210990
公开号:
AU2018210990A1
申请日:
2018.01.22
申请国别(地区):
AU
年份:
2019
代理人:
摘要:
Compositions, recombinant viruses, and recombinant viral vectors for inhibiting sarcolipin (SLN) expression or activity in a cell and for preventing or treating Duchenne Muscular Dystrophy (DMD) in a subject (e.g., a human patient having or predisposed to having DMD) and in some embodiments additionally cardiomyopathy, include a therapeutically effective amount of an inhibitor of SLN. Methods of using these compositions, recombinant viruses, and recombinant viral vectors are also described herein. These compositions, recombinant viruses, and recombinant viral vectors and methods of use provide novel therapies for DMD and associated cardiomyopathy based on the reduction of SLN expression and/or activity.
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中国工程科技知识中心
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http://www.ckcest.cn/home/

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