RNA repair for haemophilia A

Expert Rev Mol Med. 2006 Jan 10;8(1):1-8. doi: 10.1017/S1462399406010337.


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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Genetic Therapy / methods*
  • Hemophilia A / therapy*
  • Humans
  • Mice
  • RNA Precursors / genetics*
  • Spliceosomes / metabolism
  • Trans-Splicing*


  • RNA Precursors