CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B Mice

Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 loaded by vectors could induce high rates of specific site genome editing and correct disease-causing mutations. However, most monogenic genetic diseases such as hemophilia are caused by different mutations dispersed in one gene...

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Detalles Bibliográficos
Autores principales: Wang, Qingnan, Zhong, Xiaomei, Li, Qian, Su, Jing, Liu, Yi, Mo, Li, Deng, Hongxin, Yang, Yang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7393320/
https://www.ncbi.nlm.nih.gov/pubmed/32775489
http://dx.doi.org/10.1016/j.omtm.2020.06.025

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7393320/
https://www.ncbi.nlm.nih.gov/pubmed/32775489
http://dx.doi.org/10.1016/j.omtm.2020.06.025

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