title: RNA-based therapies for genodermatoses.
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 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.
authors.affiliation: Department of Dermatology, Medical Center - University of Freiburg, Freiburg, Germany.
authors.forename: Olivier
authors.initials: O
authors.lastname: Bornert
authors.affiliation: EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital of the Paracelsus Medical University, Salzburg, Austria.
authors.forename: Patricia
authors.initials: P
authors.lastname: Peking
authors.affiliation: Department of Dermatology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
authors.forename: Jeroen
authors.initials: J
authors.lastname: Bremer
authors.affiliation: EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital of the Paracelsus Medical University, Salzburg, Austria.
authors.forename: Ulrich
authors.initials: U
authors.lastname: Koller
authors.affiliation: Department of Dermatology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.|Department of Genetics, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
authors.forename: Peter C
authors.initials: PC
authors.lastname: van den Akker
authors.affiliation: Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: Annemieke
authors.initials: A
authors.lastname: Aartsma-Rus
authors.affiliation: Department of Dermatology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
authors.forename: Anna M G
authors.initials: AM
authors.lastname: Pasmooij
authors.affiliation: EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital of the Paracelsus Medical University, Salzburg, Austria.
authors.forename: Eva M
authors.initials: EM
authors.lastname: Murauer
authors.affiliation: Department of Dermatology, Medical Center - University of Freiburg, Freiburg, Germany.
authors.forename: Alexander
authors.initials: A
authors.lastname: Nyström
mesh_terms: D000818:Animals; D055785:Gene Knockdown Techniques; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D016376:Oligonucleotides, Antisense / Q000627:therapeutic use; D012313:RNA / Q000627:therapeutic use*; D012333:RNA, Messenger / Q000627:therapeutic use; D012873:Skin Diseases, Genetic / Q000628:therapy*; D020040:Trans-Splicing
chemical_list: D016376:Oligonucleotides, Antisense; D012333:RNA, Messenger; D012313:RNA
keywords: RNA trans-splicing; antisense oligonucleotides; in vitro-transcribed mRNA; siRNA; skin