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

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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Bibliographic Details
Main Authors: Qingnan Wang, Xiaomei Zhong, Qian Li, Jing Su, Yi Liu, Li Mo, Hongxin Deng, Yang Yang
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
CRISPR-Cas9
hemophilia B
targeted integration
albumin
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120301492

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http://www.sciencedirect.com/science/article/pii/S2329050120301492

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