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

Haiyan Fu, Aaron S. Meadows, Tierra Ware, Robert P. Mohney, Douglas M. McCarty

Molecular Therapy · 2017 · 16 citations · 42 references

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