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RNA repair for haemophilia A

Published online by Cambridge University Press:  10 January 2006

Hengjun Chao  and
Christopher E. Walsh
Hengjun Chao
Affiliation:
Dept of Medicine, Division of Hematology and Medical Oncology, Mt Sinai School of Medicine, One Gustave Levy Place, Box 1079, New York, NY 10029, USA.
Christopher E. Walsh
Affiliation:
Dept of Medicine, Division of Hematology and Medical Oncology, Mt Sinai School of Medicine, One Gustave Levy Place, Box 1079, New York, NY 10029, USA.
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Abstract

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.

Keywords

trans-splicingspliceosomegene transferhaemophiliaAAVRNA
Type
Review Article
Information
Expert Reviews in Molecular Medicine , Volume 8 , Issue 1 , 10 January 2006 , pp. 1 - 8
Copyright
Cambridge University Press 2006

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