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Research article

First published 2016

Guanidinoacetate Methyltransferase Deficiency

Eduardo P. Marques, Angela T. S. Wyse

Journal of Inborn Errors of Metabolism and Screening

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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 1992-11

Recombinant Human Acid α-Glucosidase Generated in Bacteria: Antigenic,but Enzymatically Inactive

Frank Martiniuk, Stephanie Tzall, Agnes Chen

DNA and Cell Biology

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Research article

First published 2019-1

Systemic Delivery of AAVB1- GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease

Allison M. Keeler, Marina Zieger, Sophia H. Todeasa [...]

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 2022-7

What Are the Trends in PCAOB Inspections and the Reported Audit Deficiencies?

Ashna L. Prasad, John C. Webster

Journal of Accounting,Auditing & Finance

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Research article

First published 2005-01

Impact of Humoral Immune Response on Distribution and Efficacy of Recombinant Adeno-Associated Virus-Derived Acid α-Glucosidase in a Model of Glycogen Storage Disease Type II

Kerry O. Cresawn, Thomas J. Fraites, Clive Wasserfall [...]

Human Gene Therapy

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Review article

First published 2019-10

Gene Therapy for Pompe Disease: The Time is now

Pasqualina Colella, Federico Mingozzi

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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Research article

First published 2001-03

Intercellular Transfer of the Virally Derived Precursor Form of Acid α-Glucosidase Corrects the Enzyme Deficiency in Inherited Cardioskeletal Myopathy Pompe Disease

Daniel F. Pauly, Thomas J. Fraites, Catalin Toma [...]

Human Gene Therapy

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