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 代理人
主权项
地址