Expert Reviews in Molecular Medicine

 


Review Article

RNA repair for haemophilia A

Hengjun Chao a1 and Christopher E. Walsh a1c1
a1 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.

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.

(Published Online January 10 2006)


Key Words: trans-splicing; spliceosome; gene transfer; haemophilia; AAV; RNA.

Correspondence:
c1 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. Tel: +1 212 241 3443; Fax: +1 212 241 4096; E-mail: christopher-e.walsh@msnyuhealth.org