Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery.

Mol Ther Methods Clin Dev · 2016 · PMC4898406 · PMID 27331076

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did not reach statistical significanceno p-value reported

In contrast, reductions in GAG in mice treated at the low dose of 1E12 vg/kg did not reach statistical significance in any tissue compared to untreated animals.

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