RNA-based therapies for genodermatoses.
Ontology highlight
ABSTRACT:
SUBMITTER: Bornert O
PROVIDER: S-EPMC5593095 | biostudies-literature | 2017 Jan
REPOSITORIES: biostudies-literature
Publications
RNA-based therapies for genodermatoses.
Bornert Olivier O Peking Patricia P Bremer Jeroen J Koller Ulrich U van den Akker Peter C PC Aartsma-Rus Annemieke A Pasmooij Anna M G AM Murauer Eva M EM Nyström Alexander A
Experimental dermatology 20170101 1
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 ...[more]
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