Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model.
Publication/Presentation Date
9-11-2020
Abstract
Mucopolysaccharidosis type IVA (MPS IVA) is due to the deficiency of GALNS (
Volume
18
First Page
50
Last Page
61
ISSN
2329-0501
Published In/Presented At
Sawamoto, K., Karumuthil-Melethil, S., Khan, S., Stapleton, M., Bruder, J. T., Danos, O., & Tomatsu, S. (2020). Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model. Molecular therapy. Methods & clinical development, 18, 50–61. https://doi.org/10.1016/j.omtm.2020.05.015
Disciplines
Medicine and Health Sciences
PubMedID
32577432
Department(s)
Fellows and Residents
Document Type
Article
COinS