ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia.

Pre-boost pMMA levels at day 55 in groups treated with Anc80 alone or combined with low-dose ImmTOR were not statistically different from post-boost levels due to high variability in baseline pMMA levels ( Figure 4 A); however, there was a clear trend toward lower pMMA levels after re-dosing in the low-dose ImmTOR-treated group (234 ± 96 μM at day 55 versus 171 ± 58 μM at day 70) compared to the vector alone group (345 ± 271 μM at day 55 versus 378 ± 257 μM at day 70).

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