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

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-modif...

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Autores principales:	Wioleta Grabowska-Pyrzewicz, Andrew Want, Jerzy Leszek, Urszula Wojda
Formato:	article
Lenguaje:	EN
Publicado:	Elsevier 2021
Materias:	Neurodegeneration Alzheimer's disease therapy gene therapy RNA therapy antisense oligonucleotide Medicine R Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/a829b7ada1024b9f994e0a86a7acb583
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spelling	oai:doaj.org-article:a829b7ada1024b9f994e0a86a7acb5832021-11-12T04:40:52ZAntisense oligonucleotides for Alzheimer's disease therapy: from the mRNA to miRNA paradigm2352-396410.1016/j.ebiom.2021.103691https://doaj.org/article/a829b7ada1024b9f994e0a86a7acb5832021-12-01T00:00:00Zhttp://www.sciencedirect.com/science/article/pii/S2352396421004850https://doaj.org/toc/2352-3964Alzheimer'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.Wioleta Grabowska-PyrzewiczAndrew WantJerzy LeszekUrszula WojdaElsevierarticleNeurodegenerationAlzheimer's diseasetherapygene therapyRNA therapyantisense oligonucleotideMedicineRMedicine (General)R5-920ENEBioMedicine, Vol 74, Iss , Pp 103691- (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Neurodegeneration Alzheimer's disease therapy gene therapy RNA therapy antisense oligonucleotide Medicine R Medicine (General) R5-920
spellingShingle	Neurodegeneration Alzheimer's disease therapy gene therapy RNA therapy antisense oligonucleotide Medicine R Medicine (General) R5-920 Wioleta Grabowska-Pyrzewicz Andrew Want Jerzy Leszek Urszula Wojda Antisense oligonucleotides for Alzheimer's disease therapy: from the mRNA to miRNA paradigm
description	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.
format	article
author	Wioleta Grabowska-Pyrzewicz Andrew Want Jerzy Leszek Urszula Wojda
author_facet	Wioleta Grabowska-Pyrzewicz Andrew Want Jerzy Leszek Urszula Wojda
author_sort	Wioleta Grabowska-Pyrzewicz
title	Antisense oligonucleotides for Alzheimer's disease therapy: from the mRNA to miRNA paradigm
title_short	Antisense oligonucleotides for Alzheimer's disease therapy: from the mRNA to miRNA paradigm
title_full	Antisense oligonucleotides for Alzheimer's disease therapy: from the mRNA to miRNA paradigm
title_fullStr	Antisense oligonucleotides for Alzheimer's disease therapy: from the mRNA to miRNA paradigm
title_full_unstemmed	Antisense oligonucleotides for Alzheimer's disease therapy: from the mRNA to miRNA paradigm
title_sort	antisense oligonucleotides for alzheimer's disease therapy: from the mrna to mirna paradigm
publisher	Elsevier
publishDate	2021
url	https://doaj.org/article/a829b7ada1024b9f994e0a86a7acb583
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_version_	1718431249545035776

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

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