Spliceosome-mediated RNA trans-splicing as a tool for gene therapy

Nat Biotechnol. 1999 Mar;17(3):246-52. doi: 10.1038/6986.

Abstract

We have developed RNA molecules capable of effecting spliceosome-mediated RNA trans-splicing reactions with a target messenger RNA precursor (pre-mRNA). Targeted trans-splicing was demonstrated in a HeLa nuclear extract, cultured human cells, and H1299 human lung cancer tumors in athymic mice. Trans-splicing between a cancer-associated pre-mRNA encoding the beta-subunit of human chorionic gonadotropin gene 6 and pre-trans-splicing molecule (PTM) RNA was accurate both in vitro and in vivo. Comparison of targeted versus nontargeted trans-splicing revealed a moderate level of specificity, which was improved by the addition of an internal inverted repeat encompassing the PTM splice site. Competition between cis- and trans-splicing demonstrated that cis-splicing can be inhibited by trans-splicing. RNA repair in a splicing model of a nonfunctional lacZ transcript was effected in cells by a PTM, which restored significant beta-galactosidase activity. These observations suggest that spliceosome-mediated RNA trans-splicing may represent a general approach for reprogramming the sequence of targeted transcripts, providing a novel approach to gene therapy.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Chorionic Gonadotropin, beta Subunit, Human / genetics
  • DNA Primers
  • Exons
  • Genetic Engineering*
  • Genetic Therapy*
  • Globins / genetics
  • HeLa Cells
  • Humans
  • Mice
  • Mice, Nude
  • Models, Biological
  • Neoplasms, Experimental
  • RNA Splicing / physiology*
  • Reverse Transcriptase Polymerase Chain Reaction
  • Sequence Analysis, DNA
  • Spliceosomes / genetics*
  • Time Factors
  • Transfection
  • Tumor Cells, Cultured
  • beta-Galactosidase / metabolism

Substances

  • Chorionic Gonadotropin, beta Subunit, Human
  • DNA Primers
  • Globins
  • beta-Galactosidase