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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...
| Autores principales: | , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2021
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| 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 |