Antisense-Oligonukleotide II

Zusammenfassung

Antisense-Oligonukleotide (ASO) sind synthetische einzelsträngige Nukleinsäuren, die an die komplementäre Sequenz der prä- mRNA oder mRNA binden, Transkription und Translation beeinflussen und damit die Proteinsynthese modulieren. Im Jahr 2016 wurden 2 ASOs zur Behandlung neuromuskulärer Erkrankungen zugelassen. In tierexperimentellen sowie klinischen Studien zeigten sich vielversprechende Wirksamkeitsnachweise von ASOs zur Behandlung weiterer neurodegenerativer Erkrankungen. In diesem Artikel sollen die gegenwärtig zur Verfügung stehenden Ergebnisse zu ASOs bei der Behandlung der Alzheimer Demenz vorgestellt werden. Basierend auf in Tierexperimenten untersuchten ASOs sollen mögliche Zielstrukturen diskutiert werden, die zukünftig für einen Einsatz der ASO-Strategie in der Behandlung psychischer Erkrankungen sprechen könnten. Darüber hinaus sollen finanzielle und ethische Aspekte, die mit der Einführung von ASOs aufkamen, vorgestellt werden.

Summary

Antisense oligonucleotides (ASO) are singlestranded nucleic acids strings that bind to a complementary sequence of the pre-mRNA or mRNA, modulate transcription and/or translation and thus, protein expression. In 2016, two ASOs were approved for treatment of neuromuscular disorders. Currently, there are further ASO drugs under research in animal models or even in clinical trials for the treatment of further neurodegenerative disorders with promising results. In this article, we present the results from studies conducted with ASO strategies for the treatment of Alzheimer`s disease. Owing to previously investigated ASOs in experimental studies, we discuss potential targets of ASO strategies and whether this approach is eligible for treatment of psychiatric disorders. Moreover, we provide an overview of the upcoming discussion regarding costs, cost-effectiveness and ethical implications of ASOs.

Schlüsselwörter

Antisense-Oligonukleotide - Alzheimer Demenz - psychische Störung

Keywords

Antisense oligonucleotides - Alzheimer disease - psychiatric disorders

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