title: RNA repair for haemophilia A.
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.
authors.affiliation: 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
authors.forename: Hengjun
authors.initials: H
authors.lastname: Chao
authors.forename: Christopher E
authors.initials: CE
authors.lastname: Walsh
mesh_terms: 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
chemical_list: D012322:RNA Precursors