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Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII

Hemophilia A is a monogenic disease with a blood clotting factor VIII (FVIII) deficiency caused by mutation in the factor VIII (F8) gene. Current and emerging treatments such as FVIII protein injection and gene therapies via AAV-delivered F8 transgene in an episome are costly and nonpermanent. Here,...

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Detalles Bibliográficos
Autores principales:	Chen, Hainan, Shi, Mi, Gilam, Avital, Zheng, Qi, Zhang, Yin, Afrikanova, Ivka, Li, Jinling, Gluzman, Zoya, Jiang, Ruhong, Kong, Ling-Jie, Chen-Tsai, Ruby Yanru
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6856096/ https://www.ncbi.nlm.nih.gov/pubmed/31727959 http://dx.doi.org/10.1038/s41598-019-53198-y

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