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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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Detalles Bibliográficos
Autores principales: Li, Qian, Su, Jing, Liu, Yi, Jin, Xiu, Zhong, Xiaomei, Mo, Li, Wang, Qingnan, Deng, Hongxin, Yang, Yang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7907211/
https://www.ncbi.nlm.nih.gov/pubmed/33718515
http://dx.doi.org/10.1016/j.omtm.2021.02.005