RNA repair for haemophilia A.
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.
Chao
Hengjun
H
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
Walsh
Christopher E
CE
D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D006467:Hemophilia A / Q000628:therapy*; D006801:Humans; D051379:Mice; D012322:RNA Precursors / Q000235:genetics*; D017381:Spliceosomes / Q000378:metabolism; D020040:Trans-Splicing
D012322:RNA Precursors