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CRISPR/Cas9 mediated somatic gene therapy for insertional mutations: the vibrator mouse model

Somatic gene therapy remains technically challenging, especially in the central nervous system (CNS). Efficiency of gene delivery, efficacy in recipient cells, and proportion of cells required for overall benefit are the key points needed to be considered in any therapeutic approach. Recent efforts...

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Detalles Bibliográficos
Autores principales:	Fu, Xin, Zhu, Jie, Duan, Yaou, Lu, Paul, Zhang, Kang
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2021
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8982555/ https://www.ncbi.nlm.nih.gov/pubmed/35693220 http://dx.doi.org/10.1093/pcmedi/pbab021

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