公开(公告)号：US20240156985A1

公开(公告)日：2024-05-16

申请号：US17985765

申请日：2022-11-11

Applicant: City University of Hong Kong

Inventor： Wenjun Xiong ,  Anh Duc Hoang ,  Baoshan Liao

IPC: A61K48/00 ,  A61K31/7105 ,  A61K38/17 ,  A61K38/46 ,  A61P27/02 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86 ,  C12N15/90

CPC classification number: A61K48/005 ,  A61K31/7105 ,  A61K38/177 ,  A61K38/465 ,  A61P27/02 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86 ,  C12N15/907 ,  C12N2310/20 ,  C12N2750/14143 ,  C12N2750/14152 ,  C12N2750/14171 ,  C12N2800/80

Abstract: Novel 5′ untranslated region (UTR)-targeting gene knock-in (KI) compositions and methods of use are disclosed. The gene KI compositions and methods exploit homology-independent targeted integration (HITI)-mediated insertion of a wild-type coding sequence (CDS) into the 5′ UTR upstream of a translation initiation element of a mutated variant of the wild-type gene. The 5′ UTR-targeting gene KI therapy compositions and methods provide safer and more efficient gene insertion compared to other gene therapy approaches.