Toward nanomedicine-enabled RNA therapeutics for Alzheimer's disease - PubMed

3 hours ago

Alzheimer's disease (AD) involves complex processes like amyloid-β aggregation, tau pathology, neuroinflammation, and neuronal loss, with current therapies being inadequate.
RNA therapeutics (e.g., ASOs, siRNAs, miRNAs, mRNAs) offer precise modulation of AD pathways but face challenges such as instability, immunogenicity, and poor blood-brain barrier (BBB) delivery.
Nanotechnology shows promise for RNA delivery in non-CNS areas but has not yet been tested in AD clinical trials, with potential to improve stability, BBB transport, and cellular targeting.
Key translational requirements include scalable manufacturing, safety assessments, immune response mitigation, and measurable brain exposure to advance nanomedicine for AD.
Future strategies involve multifunctional nanocarriers and combination RNA payloads to address AD's heterogeneity and achieve durable disease modification.

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