RNA-based therapies for genodermatoses

Olivier Bornert, Patricia Peking, Jeroen Bremer, Ulrich Koller, Peter C. van den Akker, Annemieke Aartsma-Rus, Anna M. G. Pasmooij, Eva M. Murauer, Alexander Nystroem*

*Corresponding author voor dit werk

Onderzoeksoutputpeer review

26 Citaten (Scopus)

Samenvatting

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 anti-sense 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 was to set the stage for the development of new and better therapies to improve the lives of patients and families affected by a genodermatosis.

Originele taal-2English
Pagina's (van-tot)3-10
Aantal pagina's8
TijdschriftExperimental dermatology
Volume26
Nummer van het tijdschrift1
Vroegere onlinedatum4-jul.-2016
DOI's
StatusPublished - jan.-2017

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