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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Pubblicato in:Int J Mol Sci
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2016
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Article
Accesso online: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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