Expert Reviews in Molecular Medicine: http://www.expertreviews.org/
DOI: 10.1017/S1462399406010337; 10 January 2006
Hengjun Chao and Christopher E. Walsh (2006) RNA repair for haemophilia A.
Expert Rev. Mol. Med. Vol. 8, Issue 1, DOI: 10.1017/S1462399406010337

RNA repair for haemophilia A

Hengjun Chao and Christopher E. Walsh

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The mainstay of gene transfer studies is the use of wild-type cDNAs to effect phenotypic correction of diseases. However, this strategy is not feasible for genetic diseases caused either by mutations of large genes or by dominant-negative mutations, or where the regulation of the gene is critical. In this review, we will discuss a novel RNA reprogramming strategy – spliceosome-mediated RNA trans-splicing – where the pre-messenger RNA is modified by the splicing of two independent RNA species. The use of trans-splicing to effect phenotypic change in the hereditary bleeding disorder haemophilia A will be discussed.

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Figure 1. Diagram of mRNA cis-splicing versus trans-splicing.
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Figure 2. Trans-splicing approach to repair mouse FVIII exon 16 mutation.
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