Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies.

In contrast, treatment with AAV GAA showed a trend toward reduced lysosomal area, but the difference from the untreated group was not significant.

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Mice treated with AAV GAA showed a small trend toward improvement in both measures, but it was not statistically significantly different from untreated mice.

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The 1e11 vg AAV GAA, 1e10 vg AAV α-hCD63 1 scFv:GAA, and 1e11 vg AAV α-hCD63 1 scFv:GAA groups had titers in the 10 4– 10 5 range, but these did not reach statistical significance versus control mice.

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