RNA Trans-Splicing Modulation via Antisense Molecule Interference
AbstractIn 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
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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.
Liemberger B, Piñón Hofbauer J, Wally V, Arzt C, Hainzl S, Kocher T, Murauer EM, Bauer JW, Reichelt J, Koller U. RNA Trans-Splicing Modulation via Antisense Molecule Interference. International Journal of Molecular Sciences. 2018; 19(3):762.Chicago/Turabian Style
Liemberger, Bernadette; Piñón Hofbauer, Josefina; Wally, Verena; Arzt, Claudia; Hainzl, Stefan; Kocher, Thomas; Murauer, Eva M.; Bauer, Johann W.; Reichelt, Julia; Koller, Ulrich. 2018. "RNA Trans-Splicing Modulation via Antisense Molecule Interference." Int. J. Mol. Sci. 19, no. 3: 762.
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