用于遗传性皮肤病的RNA反式剪接

RNA trans-splicing for genodermatoses.

作者信息

Bauer Johann W, Murauer Eva M, Wally Verena, Koller Ulrich

机构信息

Division of Molecular Dermatology and EB House Austria, Department of Dermatology, Paracelsus Private Medical University, Salzburg, Austria.

出版信息

Methods Mol Biol. 2013;961:441-55. doi: 10.1007/978-1-62703-227-8_30.

DOI:10.1007/978-1-62703-227-8_30

PMID:23325663

Abstract

Spliceosome-mediated RNA trans-splicing (SMaRT) is a tool that facilitates the recombination of two distinct pre-mRNA molecules. Its application for gene therapeutic purposes has been hindered by laborious procedures to identify gene-specific molecules. We have established a screening method for the identification of highly functional RNA trans-splicing molecules based on fluorescence reporters, facilitating the generation of most potent therapeutic molecules for the correction of any gene of interest.

摘要

剪接体介导的RNA反式剪接（SMaRT）是一种促进两个不同的前体mRNA分子重组的工具。其在基因治疗目的方面的应用因识别基因特异性分子的繁琐程序而受到阻碍。我们基于荧光报告基因建立了一种筛选方法，用于鉴定高功能的RNA反式剪接分子，从而有助于生成用于纠正任何感兴趣基因的最有效的治疗分子。