TY - JOUR
T1 - RNA-based therapies for genodermatoses
AU - Bornert, Olivier
AU - Peking, Patricia
AU - Bremer, Jeroen
AU - Koller, Ulrich
AU - van den Akker, Peter C.
AU - Aartsma-Rus, Annemieke
AU - Pasmooij, Anna M. G.
AU - Murauer, Eva M.
AU - Nystroem, Alexander
N1 - This article is protected by copyright. All rights reserved.
PY - 2017/1
Y1 - 2017/1
N2 - 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.
AB - 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.
KW - antisense oligonucleotides
KW - in vitro-transcribed mRNA
KW - RNA trans-splicing
KW - siRNA
KW - skin
KW - DYSTROPHIC EPIDERMOLYSIS-BULLOSA
KW - DUCHENNE MUSCULAR-DYSTROPHY
KW - PREMATURE TERMINATION CODONS
KW - DERMAL-EPIDERMAL JUNCTION
KW - SKIN ADHESION DISORDERS
KW - MODIFIED MESSENGER-RNA
KW - VII COLLAGEN
KW - GENE-THERAPY
KW - MOUSE MODEL
KW - ANTISENSE OLIGONUCLEOTIDES
U2 - 10.1111/exd.13141
DO - 10.1111/exd.13141
M3 - Review article
C2 - 27376675
SN - 0906-6705
VL - 26
SP - 3
EP - 10
JO - Experimental dermatology
JF - Experimental dermatology
IS - 1
ER -