ssODN-Mediated In-Frame Deletion with CRISPR/Cas9 Restores FVIII Function in Hemophilia A-Patient-Derived iPSCs and ECs

Given that the cDNA of F8 is too large to be packaged into adeno-associated virus (AAV) capsids, gene transfer of some versions of B-domain-deleted F8 (BDD-F8) for hemophilia A (HA) treatment has been attempted with promising results. Here, we describe an efficient gene correction via single-strande...

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Detalles Bibliográficos
Autores principales: Hu, Zhiqing, Zhou, Miaojin, Wu, Yong, Li, Zhuo, Liu, Xionghao, Wu, Lingqian, Liang, Desheng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6610636/
https://www.ncbi.nlm.nih.gov/pubmed/31261034
http://dx.doi.org/10.1016/j.omtn.2019.05.019

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6610636/
https://www.ncbi.nlm.nih.gov/pubmed/31261034
http://dx.doi.org/10.1016/j.omtn.2019.05.019

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