Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer’s disease

Molecular Therapy — Nucleic Acids 2026 Open access

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Authors: Caroline Woffindale, Natalia Galindo Riera, Matthew J. A. Wood, Miguel A. Varela
DOI: 10.1016/j.omtn.2026.102848
Cited by: 1
References: 33

Abstract

values. Functionally, treatment led to significant reductions in Aβ42 production, up to 70%, while maintaining favorable safety and specificity profiles. Collectively, our findings establish a proof of concept for multigene silencing in AD, demonstrating that rationally designed ASOs can provide robust target suppression across key pathological pathways. This strategy introduces a new paradigm in oligonucleotide design, with the potential to deliver disease-modifying benefits for patients with AD.

Fields of Study & Subjects

Alzheimer's disease research and treatments Telomeres Telomerase and Senescence Biological Research and Disease Studies

Publication details

Journal / source
Molecular Therapy — Nucleic Acids
Publisher
Elsevier BV
Volume
37
Issue
1
Pages
102848–102848
Publication date
29 January 2026
Language
EN