Sage Journals: Discover world-class research

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Research article

First published 2020-02

Endoplasmic Reticulum and Lysosomal Quality Control of Four Nonsense Mutants of Iduronate 2-Sulfatase Linked to Hunter's Syndrome

Alessandro Marazza, Carmela Galli, Elisa Fasana [...]

DNA and Cell Biology

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Research article

First published 2024-04

Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II

Shaun R. Wood, Ahsan Chaudrhy, Stuart Ellison [...]

Human Gene Therapy

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Research article

First published 2017-08

Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed,AAV9-Mediated Iduronate Sulfatase Gene Transfer

Kanut Laoharawee, Kelly M. Podetz-Pedersen, Tam T. Nguyen [...]

Human Gene Therapy

Open access

Research article

First published 2024-04

Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage

Fabio Catalano, Eva C. Vlaar, Zina Dammou [...]

Human Gene Therapy

Free access

Case report

First published 1998-5

Clinical and Morphologic Features of Mucopolysaccharidosis Type II in a Dog: Naturally Occurring Model of Hunter Syndrome

M. J. Wilkerson, D. C. Lewis, S. L. Marks [...]

Veterinary Pathology

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Research article

First published 1991-07

Multiple Sulfatase Deficiency With Early Severe Retinal Degeneration

Michael Harbord, J. Raymond Buncic, S.A. Chuang [...]

Journal of Child Neurology

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Research article

First published 1996-03

Retroviral-Mediated Transfer of the Iduronate-2-Sulfatase Gene into Lymphocytes for Treatment of Mild Hunter Syndrome (Mucopolysaccharidosis Type II). University of Minnesota Medical School,Minneapolis,Minnesota

Chester B. Whitley, R. Scott McIvor, Elena L. Aronovich [...]

Human Gene Therapy

Free access

Research article

First published 2011-8

Safety Evaluation of Chronic Intrathecal Administration of Idursulfase-IT in Cynomolgus Monkeys

Brian R. Felice, Teresa L. Wright, Robert B. Boyd [...]

Toxicologic Pathology

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Research article

First published 2025-04

Central Nervous System-Targeted Gene Therapy for the Treatment of Neurocognitive Deficits in Mucopolysaccharidosis Type II Mice

Guoqing Chen, Xia Zhan, Xiaolan Gao [...]

Human Gene Therapy

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Research article

First published 2015-06

Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II

Taichi Wakabayashi, Yohta Shimada, Kazumasa Akiyama [...]

Human Gene Therapy

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