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isolated antisense polynucleotide, pharmaceutical composition, methods for inducing an rna exon jump, to improve muscular dystrophy, to inhibit the progression of dystrophic pathology, and to improve muscle function, and, kit.
专利权人:
THE UNIVERSITY OF CHICAGO
发明人:
ELIZABETH MCNALLY
申请号:
BR112015005091
公开号:
BR112015005091A2
申请日:
2013.09.06
申请国别(地区):
BR
年份:
2017
代理人:
摘要:
Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy. One aspect of the disclosure provides an isolated antisense polynucleotide wherein the polynucleotide specifically hybridizes to an exon target region of a gamma sarcoglycan RNA, wherein the exon is selected from the group consisting of exon 4 (SEQ ID NO: 1), exon 5 (SEQ ID NO: 2), exon 6 (SEQ ID NO: 3), exon 7 (SEQ ID NO: 4) and a combination thereof. In some embodiments, the antisense polynucleotide cannot form an RNase H substrate, and in further embodiments the antisense polynucleotide comprises a modified polynucleotide backbone.
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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