公开(公告)号：US20240238394A1

公开(公告)日：2024-07-18

申请号：US18563822

申请日：2022-05-26

申请人： Modit Therapeutics Beijing Limited

发明人： Meifang ZHANG ,  Chengqi YI

IPC分类号： A61K38/52 ,  A61K31/7105 ,  A61K48/00 ,  C12N9/90 ,  C12N15/11

CPC分类号： A61K38/52 ,  A61K31/7105 ,  A61K48/005 ,  C12N9/90 ,  C12N15/11 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/531

摘要： Provided are methods, compositions, and systems for targeted pseudouridylation of RNA. In some aspects, provided are methods for editing a target RNA (e.g., mRNA) in a host cell, comprising introducing an engineered guide small nucleolar RNA (gsnoRNA) into the host cell, wherein the gsnoRNA recruits a DKC1 protein to modify a target uridine residue into a pseudouridine residue in the target RNA. In some embodiments, the DKC1 protein has cytoplasmic localization in the host cell.