AAV delivery of artificial miRNA targeting MAPT for the reduction of tau pathology in Alzheimer's disease.

Mol Ther · 2025 · PMC12703150 · PMID 40898616

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Significant, 98% tau reduction was also detectable in the CSF of high-dose amiR Tau01 animals and showed a trend toward significant reduction (95%) in high-dose amiR Tau12 animals, demonstrating that this measure can serve as a surrogate biomarker to evaluate target engagement within the brain ( Figure 3 I).

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