公开(公告)号：US12233050B2

公开(公告)日：2025-02-25

申请号：US17346036

申请日：2021-06-11

Applicant: City University of Hong Kong

Inventor： Wenjun Xiong ,  Shuyi Mai

IPC: C12N15/86 ,  A61K31/21 ,  A61K31/426 ,  A61K48/00 ,  C07H21/04 ,  C12N15/864

Abstract: A treatment of various eye conditions relating to eye enlargement is disclosed. The conditions can be treated with by inhibiting an upstream protein within the biological pathway or by increasing the expression of a downstream receptor within the same pathway. Inhibition of the upstream protein, sterol regulatory element binding protein (SREBP), has been achieved using small molecule inhibitors or nucleic acid in viral vector and increased expression of the downstream protein, bone morphogenetic protein (BMP), has been achieved by nucleic acid in viral vector. Also disclosed are the small molecule, nucleic acid and the viral vector as well as methods of treating ocular diseases.

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.