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Expert Rev Mol Med. 2006 Jan 10;8(1):1-8.

RNA repair for haemophilia A.

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Department of Medicine, Division of Hematology and Medical Oncology, Mt Sinai School of Medicine, One Gustave Levy Place, Box 1079, New York, NY 10029, USA.


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.

[Indexed for MEDLINE]

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