Antisense oligonucleotides directed against App and Rab5 normalized endosomal Rab activity and reversed DS-AD-linked degenerative phenotypes in the Dp16 mouse model of Down syndrome.

Alzheimers Dement · 2025 · PMC12058459 · PMID 40339155

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possibly significantno p-value reported

Targeting APP represents a possibly significant step toward developing precision therapies for DS‐associated dementia, potentially paving the way for more personalized and effective treatment options in this vulnerable population.

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