Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates

Published 2019 View Full Article

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Title
Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates
Authors
Keywords
α-Gal A, mRNA, Fabry, Gb3, lyso-Gb3, enzyme replacement therapy, ERT, gene therapy, lipid nanoparticles, LNPs
Journal
AMERICAN JOURNAL OF HUMAN GENETICS
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2019-03-14
DOI
10.1016/j.ajhg.2019.02.003

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