Generation of <i>GLA</i>-Knockout Human Embryonic Stem Cell Lines to Model Autophagic Dysfunction and Exosome Secretion in Fabry Disease-Associated Hypertrophic Cardiomyopathy

Generation of <i>GLA</i>-Knockout Human Embryonic Stem Cell Lines to Model Autophagic Dysfunction and Exosome Secretion in Fabry Disease-Associated Hypertrophic Cardiomyopathy

Fabry disease (FD) is a rare inherited disorder characterized by a wide range of systemic symptoms; it is particularly associated with cardiovascular and renal problems. Enzyme replacement therapy and pharmacological chaperone migalastat are the only approved and effective treatment strategies for F...

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Bibliographic Details
Main Authors: Hui-Yung Song, Chian-Shiu Chien, Aliaksandr A. Yarmishyn, Shih-Jie Chou, Yi-Ping Yang, Mong-Lien Wang, Chien-Ying Wang, Hsin-Bang Leu, Wen-Chung Yu, Yuh-Lih Chang, Shih-Hwa Chiou
Format: Article
Language:English
Published: MDPI AG 2019-04-01
Series:Cells
Subjects:
Fabry disease
human embryonic stem cells
CRISPR/Cas9 genomic editing
Mass spectrometry proteomic analysis
hypertrophic cardiomyopathy
disease model
Online Access:https://www.mdpi.com/2073-4409/8/4/327

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https://www.mdpi.com/2073-4409/8/4/327

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