申请人:University Of Southampton
公开号:EP4015648A1
公开(公告)日:2022-06-22
Disclosed herein are methods, compositions, polynucleic acid polymers, assays, and kits for inducing processing of a partially processed mRNA transcript to remove a retained intron to produce a fully processed mRNA transcript that encodes a full-length functional form of a protein. Also described herein are methods and compositions for treating a disease or condition characterized by impaired production of a full-length functional form of a protein or for treating a disease or condition characterized by a defective splicing in a subject.
本文公开了一些方法、组合物、多核苷酸聚合物、检测方法和试剂盒,用于诱导处理部分处理的 mRNA 转录本,以去除保留的内含子,从而产生完全处理的 mRNA 转录本,该转录本编码全长功能形式的蛋白质。本文还描述了用于治疗以蛋白质全长功能形式的产生受损为特征的疾病或病症,或用于治疗以受试者剪接缺陷为特征的疾病或病症的方法和组合物。