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

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Title
Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery
Authors
Keywords
MPS IIIB, global metabolomics profiling, AAV9, systemic gene delivery, biomarkers
Journal
MOLECULAR THERAPY
Volume 25, Issue 3, Pages 792-802
Publisher
Elsevier BV
Online
2017-01-29
DOI
10.1016/j.ymthe.2016.12.025

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