Rosalind Franklin University of Medicine and Science
发明人:
Michelle L. Hastings
申请号：
US15835698
公开号：
US20180117073A1
申请日:
2017.12.08
申请国别(地区):
US
年份:
2018
代理人:
摘要：
The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.
