Targeting Root Cause by Systemic scAAV9-h Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice.

No treatment is available to address the neurological need and reversibility of MPS II. We developed a scAAV9-h vector to deliver the human iduronate-2-sulfatase gene and test it in mouse model. We treated MPS II mice at different disease stages with an intravenous injection of scAAV9-mCMV-h at different doses.