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Research article

First published 2007-9

Pathology of Mucopolysaccharidosis IIIA in Huntaway Dogs

R. D. Jolly, A. C. Johnstone, E. J. Norman [...]

Veterinary Pathology

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Case report

First published 2025-7

Rare Presentation of Attenuated Mucopolysaccharidosis Type IIIA as Isolated Retinitis Pigmentosa

Josephine R. Seela, Jade Y. Moon, Sandra R. Montezuma

Journal of VitreoRetinal Diseases

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Research article

First published 2021-04

Systemic scAAV9.U1a.hSGSH Delivery Corrects Brain Biochemistry in Mucopolysaccharidosis Type IIIA at Early and Later Stages of Disease

Jennifer T. Saville, Ainslie L.K. Derrick-Roberts, Chantelle McIntyre [...]

Human Gene Therapy

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Research article

First published 2019-09

An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA

Anna L. Gray, Claire O'Leary, Aiyin Liao [...]

Human Gene Therapy

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Letter

First published 2026-4

Letter: Fluoxetine for Behavioral Problems in an 8-Year-Old with Mucopolysaccharidosis IIIA

Marta Abrantes, Tania Clemente

Journal of Child and Adolescent Psychopharmacology

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Research article

First published 2014-06

Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

Marc Tardieu, Michel Zérah, Béatrice Husson [...]

Human Gene Therapy

Open access

Case report

First published 2024

Successful use of an eye gaze AAC communication board by a young adult with advanced Sanfilippo Syndrome (MPS IIIA): Case report

Christine Brennan, Abigail Matthews, Sherri Tennant

Therapeutic Advances in Rare Disease

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Research article

First published 2016-05

A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome

Leanne K. Winner, Helen Beard, Sofia Hassiotis [...]

Human Gene Therapy

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Research article

First published 2012-12

Correction of Pathological Accumulation of Glycosaminoglycans in Central Nervous System and Peripheral Tissues of MPSIIIA Mice Through Systemic AAV9 Gene Transfer

Albert Ruzo, Sara Marcó, Miquel García [...]

Human Gene Therapy

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Research article

First published 2017-12

Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy

Haiyan Fu, Aaron S. Meadows, Ricardo J. Pineda [...]

Human Gene Therapy Clinical Development

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