Spliceosome-Mediated Trans-Splicing: The Therapeutic Cut and Paste
Spliceosome-Mediated Trans-Splicing: The Therapeutic Cut and Paste
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DOI：
10.1038/jid.2012.101
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发表时间：
2012-08-01
影响因子：
6.5
通讯作者：
Bauer, Johann W.
中科院分区：
文献类型：
--
作者：
Wally, Verena;Murauer, Eva M.;Bauer, Johann W.
剪接体介导的RNA反式剪接(SMART)是一种基于RNA的技术，用于重新编程基因以用于诊断和治疗目的。在纠正遗传病方面，SMART比传统的基因替换策略有几个优势。SMART方案最近已被用于各种遗传疾病的体外表型纠正，从大疱性表皮松解症到神经退行性疾病。体内研究目前正在将反式剪接RNA疗法推向临床应用。在这篇综述中，我们总结了SMART在医疗上的应用进展，并对其未来的应用进行了展望。《皮肤病研究杂志》(2012年)132期，1959年至1966年；doi：10.1038/jid.2012.101；2012年4月12日在线发布
Spliceosome-mediated RNA trans-splicing (SMaRT) is an RNA-based technology to reprogram genes for diagnostic and therapeutic purposes. For the correction of genetic diseases, SMaRT offers several advantages over traditional gene-replacement strategies. SMaRT protocols have recently been used for in vitro phenotypic correction of a variety of genetic disorders, ranging from epidermolysis bullosa to neurodegenerative diseases. In vivo studies are currently bringing trans-splicing RNA therapy toward clinical application. In this review, we summarize the progress made toward the medical use of SMaRT and provide an outlook on its upcoming applications. Journal of Investigative Dermatology (2012) 132, 1959-1966; doi:10.1038/jid.2012.101; published online 12 April 2012