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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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Detalles Bibliográficos
Autores principales:	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:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2016
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5187889/ https://www.ncbi.nlm.nih.gov/pubmed/27983599 http://dx.doi.org/10.3390/ijms17122089

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