Divergent Impact of Enzyme Replacement Therapy on Human Cardiomyocytes and Enterocytes Affected by Fabry Disease: Correlation with Mannose-6-phosphate Receptor Expression

Divergent Impact of Enzyme Replacement Therapy on Human Cardiomyocytes and Enterocytes Affected by Fabry Disease: Correlation with Mannose-6-phosphate Receptor Expression

Background: The impact of enzyme replacement therapy (ERT) on cardiomyocytes and intestinal cells, affected by Fabry disease (FD), is still unclear. Methods: Six patients with FD, including five family members with GLA mutation c.666delC and one with GLA mutation c.658C > T, manifesting cardiomyo...

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Bibliographic Details
Main Authors: Andrea Frustaci, Behzad Najafian, Giuseppe Donato, Romina Verardo, Cristina Chimenti, Luigi Sansone, Manuel Belli, Enza Vernucci, Matteo Antonio Russo
Format: Article
Language:English
Published: MDPI AG 2022-02-01
Series:Journal of Clinical Medicine
Subjects:
Fabry disease
GLA
globotriaosylceramide
cardiomyocyte
intestine
enzyme replacement therapy
Online Access:https://www.mdpi.com/2077-0383/11/5/1344

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