剪接体介导的RNA反式剪接在基因治疗和基因组学中的应用

M. Garcia-Blanco, M. Puttaraju, S. Mansfield, L. Mitchell
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引用次数: 14

摘要

在人类细胞中,大多数由RNA聚合酶II合成的初级转录本包含编码序列的中断,即内含子。剪接体是一种大分子酶,在称为前信使RNA剪接的过程中精确有效地去除这些内含子。这种酶也能够在一个被称为剪接体介导的RNA反式剪接的过程中重组两个反式RNA。在这篇综述中,我们重点介绍了利用这种RNA反式剪接的技术,从基因治疗到基因组学。
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Spliceosome-mediated RNA trans -splicing in gene therapy and genomics
In human cells, the majority of the primary transcripts synthesized by RNA polymerase II contain interruptions in the coding sequences, which are known as introns. The spliceosome, a macromolecular enzyme, precisely and efficiently removes these introns in a process known as pre-messenger RNA splicing. This enzyme is also capable of recombining two RNAs in trans in a process known as spliceosome-mediated RNA trans -splicing. In this review, we highlight technologies that take advantage of this RNA trans -splicing, from gene therapy to genomics.
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