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Int. J. Mol. Sci. 2018, 19(3), 762; https://doi.org/10.3390/ijms19030762

RNA Trans-Splicing Modulation via Antisense Molecule Interference

1
EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital of the Paracelsus Medical University, 5020 Salzburg, Austria
2
Laboratory for Immunological and Molecular Cancer Research, Department of Internal Medicine III with Hematology, Medical Oncology, Hemostaseology, Infectious Diseases, Rheumatology, Oncologic Center, Paracelsus Medical University, 5020 Salzburg, Austria
3
Department of Dermatology, University Hospital of the Paracelsus Medical University, 5020 Salzburg, Austria
*
Authors to whom correspondence should be addressed.
Received: 30 January 2018 / Revised: 28 February 2018 / Accepted: 5 March 2018 / Published: 7 March 2018
(This article belongs to the Special Issue Pre-mRNA Splicing 2017)
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Abstract

In recent years, RNA trans-splicing has emerged as a suitable RNA editing tool for the specific replacement of mutated gene regions at the pre-mRNA level. Although the technology has been successfully applied for the restoration of protein function in various genetic diseases, a higher trans-splicing efficiency is still desired to facilitate its clinical application. Here, we describe a modified, easily applicable, fluorescence-based screening system for the generation and analysis of antisense molecules specifically capable of improving the RNA reprogramming efficiency of a selected KRT14-specific RNA trans-splicing molecule. Using this screening procedure, we identified several antisense RNAs and short rationally designed oligonucleotides, which are able to increase the trans-splicing efficiency. Thus, we assume that besides the RNA trans-splicing molecule, short antisense molecules can act as splicing modulators, thereby increasing the trans-splicing efficiency to a level that may be sufficient to overcome the effects of certain genetic predispositions, particularly those associated with dominantly inherited diseases. View Full-Text
Keywords: RNA trans-splicing; RNA therapy; fluorescence-based screening system; antisense molecules; epidermolysis bullosa; KRT14 RNA trans-splicing; RNA therapy; fluorescence-based screening system; antisense molecules; epidermolysis bullosa; KRT14
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Liemberger, B.; Piñón Hofbauer, J.; Wally, V.; Arzt, C.; Hainzl, S.; Kocher, T.; Murauer, E.M.; Bauer, J.W.; Reichelt, J.; Koller, U. RNA Trans-Splicing Modulation via Antisense Molecule Interference. Int. J. Mol. Sci. 2018, 19, 762.

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