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Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
专利权人:
Sarepta Therapeutics; Inc.
发明人:
Schnell, Frederick Joseph
申请号:
AU2017254106
公开号:
AU2017254106A1
申请日:
2017.04.17
申请国别(地区):
AU
年份:
2018
代理人:
摘要:
The present disclosure relates to modified antisense oligonucleotides. The nucleotides described herein are of 10 to 40 nucleobases and include a targeting sequence complementary to a target region within intron 1 of a pre-mRNA of the human alpha glucosidase (GAA) gene. The target region includes at least one additional nucleobase compared to the targeting sequence, wherein the at least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional nucleobase is internal to the target region.
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中国工程科技知识中心
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