Downregulation of Mannose-6-Phosphate Receptors in Fabry Disease Cardiomyopathy: A Potential Target for Enzyme Therapy Enhancement.

Andrea FrustaciRomina VerardoRossella Scialla Giulia BagnatoMargherita VerardoMaria AlfaranoMatteo Antonio Russo

Published in: Journal of clinical medicine (2022)

M6Pr expression is remarkably reduced in FDCM as a likely result of post-translational degradation. This may explain the reduced efficacy of ERT and be a therapeutic target for the enhancement of ERT activity.

Keyphrases

poor prognosis
heart failure
replacement therapy
cell proliferation
signaling pathway
binding protein
hypertrophic cardiomyopathy
stem cells
human health
mesenchymal stem cells
left ventricular
cell therapy
atrial fibrillation