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

RNA repair for haemophilia A.

Author information

1
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. hengjun.chao@mssm.edu

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.

PMID:
16401355
DOI:
10.1017/S1462399406010337
[Indexed for MEDLINE]

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