Work

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

Year: 2026

Type: article

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.

Source: Molecular Therapy — Nucleic Acids

DOI: 10.1016/j.omtn.2026.102848

Authors: Caroline Woffindale, Natalia Galindo Riera, Matthew J.A. Wood, Miguel A. Varela

Institutions: University of Oxford, Instituto de Investigación Sanitaria La Fe, John Radcliffe Hospital

Language: English

Topic: Alzheimer's disease research and treatments

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900