1. RNA-based therapies for genodermatoses
- Subjects
RNA trans-splicing ,skin ,SKIN ADHESION DISORDERS ,VII COLLAGEN ,in vitro-transcribed mRNA ,MODIFIED MESSENGER-RNA ,GENE-THERAPY ,PREMATURE TERMINATION CODONS ,MOUSE MODEL ,ANTISENSE OLIGONUCLEOTIDES ,DUCHENNE MUSCULAR-DYSTROPHY ,siRNA ,DYSTROPHIC EPIDERMOLYSIS-BULLOSA ,antisense oligonucleotides ,DERMAL-EPIDERMAL JUNCTION - 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 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.
- Published
- 2017
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