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(13)C/(31)P 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 (13)C MRS. However, clinical implementation remains challenging due to low signal-to-noise....

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Detalles Bibliográficos
Autores principales:	Baligand, Celine, Todd, Adrian G., Lee-McMullen, Brittany, Vohra, Ravneet S., Byrne, Barry J., Falk, Darin J., Walter, Glenn A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2017
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5626920/ https://www.ncbi.nlm.nih.gov/pubmed/29018835 http://dx.doi.org/10.1016/j.omtm.2017.09.002

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