Journal
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Volume 19, Issue 3, Pages -Publisher
MDPI
DOI: 10.3390/ijms19030762
Keywords
RNA trans-splicing; RNA therapy; fluorescence-based screening system; antisense molecules; epidermolysis bullosa; KRT14
Funding
- Austrian Science Fund (FWF) [P 27336-B26]
- Paracelsus Medical University, Salzburg, Austria (PMU-FF) [A-15/01/016-KOL]
- DEBRA Austria
- Austrian Science Fund (FWF) [P27336] Funding Source: Austrian Science Fund (FWF)
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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.
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