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May 11, 2026The Journal of Prevention of Alzheimer s Disease2 citationsOpen Access

RNA-based therapeutics for Alzheimer’s disease and related tauopathies: challenges and opportunities

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BRBinita RajbanshiIBIlaria BrentariMDMichela A. Denti

Key Points

  • This review aims to explore RNA-based therapeutic strategies for targeting tau accumulation in tauopathies.
  • Investigated diverse RNA modalities such as mRNA, ASO, RNAi, and SSO.
  • Outlined translational barriers, clinical trials, and patent trends related to RNA therapeutics.
  • Evaluated the challenges and opportunities presented by RNA-based therapies.
  • RNA-based therapies show promise in targeting the molecular origin of tau.
  • Clinical trials for traditional tau therapies have often failed, indicating a need for alternative approaches.
  • Emerging modalities face unique challenges but also present significant opportunities for disease modification.

Abstract

Tauopathies are neurodegenerative diseases characterized by pathological tau protein accumulation. Though therapies involving monoclonal antibodies and small-molecule inhibitors have progressed, they have so far failed in multiple clinical trials, underscoring the need for innovative molecular approaches. RNA-based therapies offer an alternative disease-modifying approach by being able to target tau at its molecular origin. Diverse modalities, such as mRNA, ASO, RNAi, and SSO, offer distinct promises. Though their challenges are equally diverse, they also share common problems. This review examines the nascent field of RNA therapeutics for tauopathies, outlining emerging modalities, translational barriers, molecular targets, clinical trials, and patent trends.

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Cite This Study

Rajbanshi et al. (2026) studied this question.

synapsesocial.com/papers/6a0171983a9f334c28271b1ahttps://doi.org/10.1016/j.tjpad.2026.100585
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