CAG-targeted brain-permeable therapy tested in biallelic humanized polyQ mouse models.

Mol Ther Nucleic Acids · 2025 · PMC11960632 · PMID 40171277

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barely significantno p-value reported

In contrast to HD mice, the lowering effects of A4(P10A) and A4(P10,11A) shReagents on mutant ATXN3 levels in Ki 150Q/21Q mice were only barely significant and showed no clear association with the transduction efficiency of brain structures ( Figures 3 B and 3C).

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may be significantno p-value reported

The level of transduction of a brain region may be significant but insufficient for protein lowering, which may also depend on the types of cells containing polyQ protein (cell populations transduced).

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