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ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHODS OF TREATING DYSTROPHIES
专利权人:
发明人:
MCNALLY ELIZABETH
申请号:
IN2460/DELNP/2015
公开号:
IN2015DN02460A
申请日:
2015.03.26
申请国别(地区):
IN
年份:
2016
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 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.
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中国工程科技知识中心
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