Advances in AAV-CRISPR/Cas9-Mediated Hemophilia A Gene Therapy --Review / 中国实验血液学杂志
Journal of Experimental Hematology
; (6): 1890-1893, 2023.
Article
em Zh
| WPRIM
| ID: wpr-1010055
Biblioteca responsável:
WPRO
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.
Palavras-chave
Texto completo:
1
Índice:
WPRIM
Assunto principal:
Terapia Genética
/
Hemofilia B
/
Sistemas CRISPR-Cas
/
Edição de Genes
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Vetores Genéticos
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Hemofilia A
Limite:
Humans
Idioma:
Zh
Revista:
Journal of Experimental Hematology
Ano de publicação:
2023
Tipo de documento:
Article