Ionis Pharmaceuticals, Inc.;Rosalind Franklin University of Medicine and Science
发明人:
申请号：
EP14867872.5
公开号：
EP3077510A4
申请日:
2014.12.02
申请国别(地区):
EP
年份:
2017
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.
