Domain-substituted IGF2 tag modulates targeting of lentiviral gene therapy for Hunter syndrome.

These findings indicate that IDS.SWAP variants and IDS.IGF2 achieved similar efficacy in the brain, showed a trend towards superior efficacy on histopathology markers compared to IDS.IGF2del in some brain areas and showed clearly enhanced efficacy compared to IDSco, as evidenced by greater reductions in cerebral heparan sulfate, alleviation of astrogliosis, and enhanced LAMP1 clearance.

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These results demonstrate that gene therapy with SWAP vectors effectively prevented alcian blue and LAMP1-related pathology in liver, spleen and cardiac muscle, and nearly fully restored those bone microarchitecture parameters that were significantly different between Ids y/ − and wild-type mice, although these improvements did not reach statistical significance.

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