Generation of GLA-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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Pubblicato in:Cells
Autori principali: Song, Hui-Yung, Chien, Chian-Shiu, Yarmishyn, Aliaksandr A., Chou, Shih-Jie, Yang, Yi-Ping, Wang, Mong-Lien, Wang, Chien-Ying, Leu, Hsin-Bang, Yu, Wen-Chung, Chang, Yuh-Lih, Chiou, Shih-Hwa
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6523555/
https://ncbi.nlm.nih.gov/pubmed/30965672
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/cells8040327
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