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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

Retricted access

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 2016-11

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice

Christian Hinderer, Nathan Katz, Jean-Pierre Louboutin [...]

Human Gene Therapy

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Research article

First published 1996-02

Preclinical Studies of Lymphocyte Gene Therapy for Mild Hunter Syndrome (Mucopolysaccharidosis Type II)

Stephen E. Braun, Dao Pan, Elena L. Aronovich [...]

Human Gene Therapy

Open access

Research article

First published 2022-12

Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells

Miles C. Smith, Lalitha R. Belur, Andrea D. Karlen [...]

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

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

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Research article

First published 2023-01

Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction

Kelly M. Podetz-Pedersen, Kanut Laoharawee, Sajya Singh [...]

Human Gene Therapy

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Research article

First published 2026-2

Intravenous Delivery of a Gene Therapy Vector that Expresses an Antitransferrin Receptor 1 Nanobody-I2S Fusion Protein Rescued Central Nervous System Lysosomal Burden in Mucopolysaccharidosis II Mice

Wanida Ruangsiriluk, Sarah Melissa P. Jacobo, Andrea Iskenderian [...]

Human Gene Therapy

Open access

Review article

First published 2018

Hematopoietic Stem Cell Transplantation in Mucopolysaccharidosis Type II

Anneliese L. Barth, Dafne D. G. Horovitz

Journal of Inborn Errors of Metabolism and Screening

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