Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery

Mucopolysaccharidosis (MPS) IIIB is a lysosomal storage disease with complex CNS and somatic pathology due to a deficiency in α-N-acetylglucosaminidase (NAGLU). Using global metabolic profiling by mass spectrometry targeting 361 metabolites, this study detected significant decreases in 225 and incre...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Fu, Haiyan, Meadows, Aaron S., Ware, Tierra, Mohney, Robert P., McCarty, Douglas M.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5363204/
https://ncbi.nlm.nih.gov/pubmed/28143737
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2016.12.025
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