- 专利标题: COMPOSITIONS, SYSTEMS AND METHODS OF RNA EDITING USING DKC1
-
申请号: US18563822申请日: 2022-05-26
-
公开(公告)号: US20240238394A1公开(公告)日: 2024-07-18
- 发明人: Meifang ZHANG , Chengqi YI
- 申请人: Modit Therapeutics Beijing Limited
- 申请人地址: CN Beijing
- 专利权人: Modit Therapeutics Beijing Limited
- 当前专利权人: Modit Therapeutics Beijing Limited
- 当前专利权人地址: CN Beijing
- 优先权: WO TCN2021096122 2021.05.26
- 国际申请: PCT/CN2022/095172 2022.05.26
- 进入国家日期: 2023-11-22
- 主分类号: A61K38/52
- IPC分类号: A61K38/52 ; A61K31/7105 ; A61K48/00 ; C12N9/90 ; C12N15/11
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.
信息查询
IPC分类: