In vivo PCSK9 gene editing using an all-in-one self-cleavage AAV-CRISPR system

In vivo PCSK9 gene editing using an all-in-one self-cleavage AAV-CRISPR system

Adeno-associated virus (AAV)-mediated delivery of the clustered regularly interspaced short palindromic repeat-CRISPR-associated protein 9 (CRISPR-Cas9) has shown promising results in preclinical models. However, the long-term expression of Cas9 mediated by AAV in the post-mitotic cells raises conce...

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Bibliographic Details
Main Authors: Qian Li, Jing Su, Yi Liu, Xiu Jin, Xiaomei Zhong, Li Mo, Qingnan Wang, Hongxin Deng, Yang Yang
Format: Article
Language:English
Published: Elsevier 2021-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
CRISPR-Cas9
PCSK9
self-cleavage
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000206

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http://www.sciencedirect.com/science/article/pii/S2329050121000206

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