Liver targeted AAV8 gene therapy ameliorates skeletal and cardiovascular pathology in mucopolysaccharidosis IVA murine model

Published 2020 View Full Article

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Title
Liver targeted AAV8 gene therapy ameliorates skeletal and cardiovascular pathology in mucopolysaccharidosis IVA murine model
Authors
Keywords
MPS IVA, GALNS, AAV, liver targeting, keratan sulfate, skeletal dysplasia, cardiovascular disease
Journal
Molecular Therapy-Methods & Clinical Development
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2020-05-22
DOI
10.1016/j.omtm.2020.05.015

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