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OLIGOMÈRES ANTISENS ET PROCÉDÉS D'UTILISATION DE CEUX-CI POUR LE TRAITEMENT DE MALADIES ASSOCIÉES AU GÈNE DE L'ALPHA-GLUCOSIDASE ACIDE
专利权人:
Sarepta Therapeutics; Inc.
发明人:
SCHNELL, Frederick, Joseph
申请号:
EP17786423
公开号:
EP3445405A4
申请日:
2017.04.17
申请国别(地区):
EP
年份:
2019
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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