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Forschungsdatenbank PMU-SQQUID

RNA-based therapies for genodermatoses.
Bornert, O; Peking, P; Bremer, J; Koller, U; van den Akker, PC; Aartsma-Rus, A; Pasmooij, AM; Murauer, EM; Nyström, A;
Exp Dermatol. 2017; 26(1):3-10
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PMU-Autor/inn/en

Ebner Patricia
Koller Ulrich
Murauer Eva Maria

Abstract

Genetic disorders affecting the skin, genodermatoses, constitute a large and heterogeneous group of diseases, for which treatment is generally limited to management of symptoms. RNA-based therapies are emerging as a powerful tool to treat genodermatoses. In this review, we discuss in detail RNA splicing-modulation by antisense oligonucleotides and RNA trans-splicing, transcript-replacement and genome editing by in vitro transcribed mRNAs, and gene knockdown by small interfering RNA and antisense oligonucleotides. We present the current state of these therapeutic approaches and critically discuss their opportunities, limitations and the challenges that remain to be solved. The aim of this review is to set the stage for the development of new and better therapies to improve the lives of patients and families affected by a genodermatosis. This article is protected by copyright. All rights reserved. This article is protected by copyright. All rights reserved.


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antisense oligonucleotides
in vitro-transcribed mRNA
RNA trans-splicing
siRNA
skin