Yissum Research Development Company of the Hebrew University of Jerusalem Ltd.
发明人:
Batsheva KEREM,Michal TUR SINAI,Loren PRICE,Stephen Donald WILTON,Sue FLETCHER
申请号：
US15869664
公开号：
US20180155723A1
申请日:
2018.01.12
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Provided are oligonucleotides capable of binding to and modulating the splicing of the pre-mRNA of the CFTR gene, compositions including said oligonucleotides, kits including the compositions, and uses thereof. In particular, the subject matter provides compositions of oligonucleotides useful in methods for suppressing exon skipping optionally in combination with additional CFTR therapeutics.
