Rosalind Franklin University of Medicine and Science;Ionis Pharmaceuticals, Inc.
发明人:
Frank Rigo,Michelle L. Hastings
申请号：
US16536981
公开号：
US20200216846A1
申请日:
2019.08.09
申请国别(地区):
US
年份:
2020
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.
