Advances in AAV-CRISPR/Cas9-Mediated Hemophilia A Gene Therapy --Review.
10.19746/j.cnki.issn.1009-2137.2023.06.045
- Author:
Shuai FANG
1
;
Gang WANG
2
;
Lin-Hua YANG
3
Author Information
1. Department of Biochemistry and Molecular Biology, School of Basic Medicine, Shanxi Medical University.
2. Department of Hematology, The Second Hospital of Shanxi Medical University, Taiyuan 030001, Shanxi Province, China.
3. Department of Hematology, The Second Hospital of Shanxi Medical University, Taiyuan 030001, Shanxi Province, China. E-mail:Yanglh5282@163.com.
- Publication Type:Journal Article
- Keywords:
AAV vector;
gene therapy;
hemophilia A;
CRISPR-Cas9;
expression target sites
- MeSH:
Humans;
Hemophilia A/therapy*;
CRISPR-Cas Systems;
Hemophilia B/therapy*;
Gene Editing;
Genetic Therapy;
Genetic Vectors
- From:
Journal of Experimental Hematology
2023;31(6):1890-1893
- CountryChina
- Language:Chinese
-
Abstract:
Hemophilia A(HA) is an X-linked recessive bleeding disorder caused by mutations in coagulation factor VIII. Nowadays, exogenous coagulation factor replacement therapy is the main treatment. With the continuous development of gene therapy, new research directions have been provided for the treatment of hemophilia A. CRISPR-Cas9 technology was applied to select suitable target sites, and mediate the targeted knock-in and efficient expression of exogenous B-domain-deleted FⅧ variant gene through corresponding vectors for the treatment of hemophilia A.CRISPR-Cas9 technology is an emerging gene editing tool with great efficiency, safety and effectiveness, and has been widely used in hemophilia gene therapy research. This paper reviews the vector selection, construction of therapeutic genes, gene editing technology and selection of expression target sites for hemophilia A gene therapy at this stage.
