(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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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Baligand, Celine, Todd, Adrian G., Lee-McMullen, Brittany, Vohra, Ravneet S., Byrne, Barry J., Falk, Darin J., Walter, Glenn A.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
Assuntos:
Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5626920/
https://ncbi.nlm.nih.gov/pubmed/29018835
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.09.002
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