发明名称 |
ANTISENSE-INDUCED EXON2 INCLUSION IN ACID ALPHA-GLUCOSIDASE |
摘要 |
The present disclosure relates to antisense oligomers and related compositions and methods for inducing exon inclusion as a treatment for glycogen storage disease type II (GSD-II) (also known as Pompe disease, glycogenosis II, acid maltase deficiency (AMD), acid alpha-glucosidase deficiency, and lysosomal alpha-glucosidase deficiency), and more specifically relates to inducing inclusion of exon 2 and thereby restoring levels of enzymatically active acid alpha-glucosidase (GAA) protein encoded by the GAA gene. |
申请公布号 |
WO2015035231(A1) |
申请公布日期 |
2015.03.12 |
申请号 |
WO2014US54384 |
申请日期 |
2014.09.05 |
申请人 |
SAREPTA THERAPEUTICS, INC.;MURDOCH UNIVERSITY |
发明人 |
WILTON, STEPHEN DONALD;FLETCHER, SUE;HANSON, GUNNAR JAMES;BESTWICK, RICHARD KEITH |
分类号 |
C12N15/113;A61K31/712;A61K31/7125;C07H21/00 |
主分类号 |
C12N15/113 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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