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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...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2021
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Materias: | |
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 |