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1-5 of 5 results for SUMF1

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Select article: Multiple Sulfatase Deficiency: A Case Series With a Novel Mutation

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Research article

First published 2018-11

Multiple Sulfatase Deficiency: A Case Series With a Novel Mutation

Leen Hijazi, Amna Kashgari, Majid Alfadhel

Journal of Child Neurology

Select article: 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

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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

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

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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

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

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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

Select article: Safety of Arylsulfatase A Overexpression for Gene Therapy of Metachromatic Leukodystrophy

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Research article

First published 2007-09

Safety of Arylsulfatase A Overexpression for Gene Therapy of Metachromatic Leukodystrophy

A. Capotondo, M. Cesani, S. Pepe [...]

Human Gene Therapy

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