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Article

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

9 Feb 2021

Abstract excerpt

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 concerns with specificity and immunogenicity. Thus, it would be advantageous to limit the duration of Cas9 expression...

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PMID: 33718515
DOI: 10.1016/j.omtm.2021.02.005

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