RNA-based therapies for genodermatoses #MMPMID27376675
Bornert O; Peking P; Bremer J; Koller U; van den Akker PC; Aartsma-Rus A; Pasmooij AMG; Murauer EM; Nyström A
Exp Dermatol 2017[Jan]; 26 (1): 3-10 PMID27376675show ga
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 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.