4.7 Article

Antisense oligonucleotides for Alzheimer's disease therapy: from the mRNA to miRNA paradigm

Journal

EBIOMEDICINE
Volume 74, Issue -, Pages -

Publisher

ELSEVIER
DOI: 10.1016/j.ebiom.2021.103691

Keywords

Neurodegeneration; therapy; gene therapy; RNA therapy; antisense oligonucleotide; miRNA; RNA delivery; preclinical testing; clinical trials; Alzheimer's disease

Funding

  1. European Union's Horizon 2020 FET OPEN [737390]
  2. Polish Ministry of Science and Higher Education [3548/H2020/COFUND/2016/2]
  3. Polish National Science Centre [OPUS 2018/29/B/NZ7/02757]

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Alzheimer's disease poses a therapeutic challenge due to its complex etiology, with recent progress in ASO-based approaches showing promising potential for multi-targeted therapy.
Alzheimer's disease (AD) represents a particular therapeutic challenge because its aetiology is very complex, with dynamic progression from preclinical to clinical stages. Several potential therapeutic targets and strategies were tested for AD, in over 2000 clinical trials, but no disease-modifying therapy exists. This failure indicates that AD, as a multifactorial disease, may require multi-targeted approaches and the delivery of therapeutic molecules to the right place and at the right disease stage. Opportunities to meet the challenges of AD therapy appear to come from recent progress in knowledge and methodological advances in the design, synthesis, and targeting of brain mRNA and microRNA with synthetic antisense oligonucleotides (ASOs). Several types of ASOs allow the utilisation of different mechanisms of posttranscriptional regulation and offer enhanced effects over alternative therapeutics. This article reviews ASO-based approaches and targets in preclinical and clinical trials for AD, and presents the future perspective on ASO therapies for AD. (C) 2021 The Authors. Published by Elsevier B.V.

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