ArticleMolecular therapy. Nucleic acids2026
Design, validation, and functional impact of oligonucleotides for multigene silencing in Alzheimer's disease.
Article in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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1 citing paper in PubMed.
- To scramble or not: Steric blocking 2'-MOE/PS control antisense oligonucleotides across cellular models.Molecular therapy. Nucleic acids · 2026Article
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Abstract
Alzheimer's disease (AD) is characterized by overlapping pathological processes, including amyloid-beta (Aβ) accumulation, tau hyperphosphorylation, mitochondrial dysfunction, and neuroinflammation. Monogenic therapies have shown limited benefits, and only in a subset of patients, as other pathological processes continue to drive disease progression. Given the multifactorial and heterogeneous nature of AD, therapeutics targeting more than one gene simultaneously represent a promising strategy to achieve broader therapeutic outcomes. This study highlights the advantages of multigene RNA-based therapeutics, which may overcome compensatory mechanisms and patient heterogeneity. Here, we report the design and functional validation of antisense oligonucleotides (ASOs) specifically engineered for simultaneous silencing of more than one AD-related gene. Using algorithm-assisted sequence design, we generated 11 bispecific gapmer ASOs from 20 candidate genes. In human and mouse cellular models, these ASOs achieved potent and sustained knockdown with picomolar to low-nanomolar IC
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