EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital of the Paracelsus Medical University, 5020 Salzburg, Austria.
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.
Int J Mol Sci. 2018 Mar 7;19(3):762. doi: 10.3390/ijms19030762.
In recent years, RNA -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 -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 -specific RNA -splicing molecule. Using this screening procedure, we identified several antisense RNAs and short rationally designed oligonucleotides, which are able to increase the -splicing efficiency. Thus, we assume that besides the RNA -splicing molecule, short antisense molecules can act as splicing modulators, thereby increasing the -splicing efficiency to a level that may be sufficient to overcome the effects of certain genetic predispositions, particularly those associated with dominantly inherited diseases.
近年来，RNA 剪接已成为一种合适的 RNA 编辑工具，可在 pre-mRNA 水平特异性替换突变基因区域。尽管该技术已成功应用于恢复各种遗传疾病中的蛋白质功能，但仍需要更高的剪接效率来促进其临床应用。在这里，我们描述了一种改良的、易于应用的荧光基础筛选系统，用于生成和分析专门能够提高选定 RNA 剪接分子 RNA 重编程效率的反义分子。使用这种筛选程序，我们鉴定了几种反义 RNA 和短的合理设计的寡核苷酸，它们能够提高剪接效率。因此，我们假设除了 RNA 剪接分子之外，短的反义分子可以作为剪接调节剂，从而将剪接效率提高到足以克服某些遗传易感性的程度，特别是那些与显性遗传疾病相关的程度。
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