Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome

Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome

Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder caused by deficiency of the iduronate-2-sulfatase (IDS) enzyme, resulting in cellular accumulation of glycosaminoglycans (GAGs) throughout the body. Treatment of MPS II remains a considerable challenge as current enzyme replaceme...

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Bibliographic Details
Main Authors: Annie Arguello, René Meisner, Elliot R. Thomsen, Hoang N. Nguyen, Ritesh Ravi, Jeffrey Simms, Iris Lo, Jessica Speckart, Julia Holtzman, Thomas M. Gill, Darren Chan, Yuhsiang Cheng, Chi-Lu Chiu, Jason C. Dugas, Meng Fang, Isabel A. Lopez, Hilda Solanoy, Buyankhishig Tsogtbaatar, Yuda Zhu, Akhil Bhalla, Kirk R. Henne, Anastasia G. Henry, Anthony Delucchi, Simona Costanzo, Jeffrey M. Harris, Dolores Diaz, Kimberly Scearce-Levie, Pascal E. Sanchez
Format: Article
Language:English
Published: American Society for Clinical investigation 2021-10-01
Series:JCI Insight
Subjects:
Neuroscience
Therapeutics
Online Access:https://doi.org/10.1172/jci.insight.145445

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