13C/31P MRS Metabolic Biomarkers of Disease Progression and Response to AAV Delivery of hGAA in a Mouse Model of Pompe Disease

13C/31P MRS Metabolic Biomarkers of Disease Progression and Response to AAV Delivery of hGAA in a Mouse Model of Pompe Disease

The development of therapeutic clinical trials for glycogen storage disorders, including Pompe disease, has called for non-invasive and objective biomarkers. Glycogen accumulation can be measured in vivo with 13C MRS. However, clinical implementation remains challenging due to low signal-to-noise. O...

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Bibliographic Details
Main Authors: Celine Baligand, Adrian G. Todd, Brittany Lee-McMullen, Ravneet S. Vohra, Barry J. Byrne, Darin J. Falk, Glenn A. Walter
Format: Article
Language:English
Published: Elsevier 2017-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene therapy
MR spectroscopy
Pompe disease
glycogen storage disorder
MRI
AAV
glucose-6-phosphate
glycogen
carbon
phosphorus
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050117300992

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http://www.sciencedirect.com/science/article/pii/S2329050117300992

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