CRISPR/Cas9-Mediated in vivo Genetic Correction in a Mouse Model of Hemophilia A

Hemophilia A (HA), a common bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII), has long been considered an attractive target for gene therapy studies. However, full-length F8 cDNA cannot be packaged efficiently by adeno-associated virus (AAV) vectors. As the second most pre...

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Detalles Bibliográficos
Autores principales: Luo, Sanchuan, Li, Zhongxiang, Dai, Xin, Zhang, Rui, Liang, Zhibing, Li, Wenzhou, Zeng, Ming, Su, Jinfeng, Wang, Jun, Liang, Xia, Wu, Yong, Liang, Desheng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Cell and Developmental Biology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8415270/
https://www.ncbi.nlm.nih.gov/pubmed/34485274
http://dx.doi.org/10.3389/fcell.2021.672564

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