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First published 2010-8

Screening of Late-Onset Pompe Disease in a Sample of Mexican Patients With Myopathies of Unknown Etiology: Identification of a Novel Mutation in the Acid α-Glucosidase Gene

Miguel Angel Alcántara-Ortigoza, Ariadna González-del Angel, Rehotbevely Barrientos-Ríos [...]

Journal of Child Neurology

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Research article

First published 2001-05

Long-Term Efficacy after [E1 -,polymerase - ] Adenovirus-Mediated Transfer of Human Acid-α-Glucosidase Gene into Glycogen Storage Disease Type II Knockout Mice

E.Y. Ding, B.L. Hodges, H. Hu [...]

Human Gene Therapy

Free access

Research article

First published 2005-1

High-resolution Light Microscopy (HRLM) and Digital Analysis of Pompe Disease Pathology

Colleen M. Lynch, Jennifer Johnson, Charles Vaccaro [...]

Journal of Histochemistry & Cytochemistry

Open access

Review article

First published 2020

Advancements in AAV-mediated Gene Therapy for Pompe Disease

S.M. Salabarria, J. Nair, N. Clement [...]

Journal of Neuromuscular Diseases

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Research article

First published 2012-3

Infantile Pompe Disease: Clinical and Genetic Characteristics With an Experience of Enzyme Replacement Therapy

Anna Cho, Su Jin Kim, Byung Chan Lim [...]

Journal of Child Neurology

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Research article

First published 2010-12

p.D645E of Acid α-Glucosidase Is the Most Common Mutation in Thai Patients with Infantile-Onset Pompe Disease

Pramuk Amarinthnukrowh, Siraprapa Tongkobpetch, Apichai Kongpatanayothin [...]

Genetic Testing and Molecular Biomarkers

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Research article

First published 2019-07

Salmeterol with Liver Depot Gene Therapy Enhances the Skeletal Muscle Response in Murine Pompe Disease

Sang-oh Han, Songtao Li, Jeffrey I. Everitt [...]

Human Gene Therapy

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Research article

First published 2015-11

Synergistic Efficacy from Gene Therapy with Coreceptor Blockade and a β 2 -Agonist in Murine Pompe Disease

Sang-oh Han, Songtao Li, Andrew Bird [...]

Human Gene Therapy

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Research article

First published 1997-09

Retroviral Transfer of Acid α -Glucosidase cDNA to Enzyme-Deficient Myoblasts Results in Phenotypic Spread of the Genotypic Correction by Both Secretion and Fusion

Joseph Z. Zaretsky, Fabio Candotti, Cornelius Boerkoel [...]

Human Gene Therapy

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Research article

First published 2015-01

Enhanced Efficacy from Gene Therapy in Pompe Disease Using Coreceptor Blockade

Sang-oh Han, Songtao Li, Elizabeth D. Brooks [...]

Human Gene Therapy

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