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

Internet

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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