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Gene Therapy Progress and Prospects: Reprograming gene expression by trans-splicing

Gene Therapy volume 12, pages 1477–1485 (2005)Cite this article

Abstract

The term ‘trans-splicing’ encompasses several platform technologies that combine two RNA or protein molecules to generate a new, chimeric product. RNA trans-splicing reprograms the sequences of endogenous messenger mRNA or pre-mRNA, converting them to a new, desired gene product. Trans-splicing has broad applications, depending on the nature of the sequences that are inserted or trans-spliced to the defined target. Trans-splicing RNA therapy offers significant advantages over conventional gene therapy: expression of the trans-spliced sequence is controlled by the endogenous regulation of the target pre-mRNA; reduction or elimination of undesirable ectopic expression; the ability to use smaller constructs that trans-splice only a portion of the gene to be replaced; and the conversion of dominant-negative mutations to wild-type gene products.

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  1. Intronn Inc, Gaithersburg, MD, USA

    L G Mitchell & G J McGarrity

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  1. L G Mitchell
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  2. G J McGarrity
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Mitchell, L., McGarrity, G. Gene Therapy Progress and Prospects: Reprograming gene expression by trans-splicing. Gene Ther 12, 1477–1485 (2005). https://doi.org/10.1038/sj.gt.3302596

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