发明名称 |
ANTISENSE COMPOUNDS AND USES THEREOF |
摘要 |
The present invention provides compounds comprising oligonucleotides complementary to a CLN3 transcript. Certain such compounds are useful for hybridizing to a CLN3 transcript, including but not limited to a CLN3 transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CLN3 transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Batten Disease. |
申请公布号 |
EP3077510(A2) |
申请公布日期 |
2016.10.12 |
申请号 |
EP20140867872 |
申请日期 |
2014.12.02 |
申请人 |
IONIS PHARMACEUTICALS, INC.;ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE |
发明人 |
RIGO, FRANK;HASTINGS, MICHELLE, L. |
分类号 |
C12N15/113 |
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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