Using CRISPR/Cas9-Mediated GLA Gene Knockout as an In Vitro Drug Screening Model for Fabry Disease

The CRISPR/Cas9 Genome-editing system has revealed promising potential for generating gene mutation, deletion, and correction in human cells. Application of this powerful tool in Fabry disease (FD), however, still needs to be explored. Enzyme replacement therapy (ERT), a regular administration of re...

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Detalhes bibliográficos
Publicado no:Int J Mol Sci
Main Authors: Song, Hui-Yung, Chiang, Huai-Chih, Tseng, Wei-Lien, Wu, Ping, Chien, Chian-Shiu, Leu, Hsin-Bang, Yang, Yi-Ping, Wang, Mong-Lien, Jong, Yuh-Jyh, Chen, Chung-Hsuan, Yu, Wen-Chung, Chiou, Shih-Hwa
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5187889/
https://ncbi.nlm.nih.gov/pubmed/27983599
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms17122089
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