ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE; INC.;IONIS PHARMACEUTICALS, INC.;IONIS PHARMACEUTICALS
发明人:
RIGO, FRANK,HASTINGS, MICHELLE L.
申请号：
CA2931829
公开号：
CA2931829A1
申请日:
2014.12.02
申请国别(地区):
CA
年份:
2015
代理人:
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.
