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Compositions and methods for correcting limb girdle muscular dystrophy type 2C using exon skipping
专利权人:
THE UNIVERSITY OF CHICAGO;NORTHWESTERN UNIVERSITY
发明人:
Elizabeth McNally,Eugene Wyatt
申请号:
US16395741
公开号:
US10801029B2
申请日:
2019.04.26
申请国别(地区):
US
年份:
2020
代理人:
摘要:
The invention is directed to one or more antisense polynucleotides and their use in pharmaceutical compositions in a strategy to induce exon skipping in the γ-sarcoglycan gene in patients suffering from Limb-Girdle Muscular Dystrophy-2C (LGM-D2C) or in patients at risk of such a disease. The invention also provides methods of preventing or treating muscular dystrophy. e.g., LGMD2C, by exon skipping in the gamma sarcoglycan gene using antisense polynucleotides. Accordingly, in some aspects the invention provides an isolated antisense oligonucleotide, wherein the oligonucleotide specifically hybridizes to an exon target region of a γ-sarcoglycan RNA. In another aspect, the invention provides a method of inducing exon-skipping of a gamma sarcoglycan RNA, comprising delivering an antisense oligonucleotide or a composition to a cell.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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