申请人:Rosalind Franklin University of Medicine and Science
公开号:US11116785B2
公开(公告)日:2021-09-14
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.
本公开总体上涉及包含与囊性纤维化跨膜传导调节剂(CFTR)RNA 转录本互补的寡核苷酸的化合物。某些此类化合物可用于与 CFTR RNA 转录本杂交,包括但不限于细胞中的 CFTR RNA 转录本。在某些实施方案中,这种杂交可调节 CFTR 转录本的剪接。在某些实施方案中,此类化合物用于治疗与囊性纤维化相关的一种或多种症状。