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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Autori principali:	Celine Baligand, Adrian G. Todd, Brittany Lee-McMullen, Ravneet S. Vohra, Barry J. Byrne, Darin J. Falk, Glenn A. Walter
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2017-12-01
Serie:	Molecular Therapy: Methods & Clinical Development
Soggetti:	gene therapy MR spectroscopy Pompe disease glycogen storage disorder MRI AAV glucose-6-phosphate glycogen carbon phosphorus
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2329050117300992
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