Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing.

Journal Article (Journal Article)

The functional repertoire of the human genome is amplified by the differential assortment of exons. Spliceosome-mediated RNA trans-splicing can mobilize these packets of genetic information to reprogram mRNAs. In principle, this process could repair defective transcripts in loss-of-function genetic disorders in humans. We developed a tractable lacZ repair system to serve as a model for these genetic disorders. Targeted pre-trans-splicing RNA molecules efficiently and specifically repaired mutated lacZ transcripts and restored enzymatic activity in human cells. The development of this model confirms the potential for spliceosome-mediated RNA trans-splicing in genetic repairs and provides a powerful tool for rational design and in vitro evolution of pre-trans-splicing molecules.

Full Text

Duke Authors

Cited Authors

  • Puttaraju, M; DiPasquale, J; Baker, CC; Mitchell, LG; Garcia-Blanco, MA

Published Date

  • August 2001

Published In

Volume / Issue

  • 4 / 2

Start / End Page

  • 105 - 114

PubMed ID

  • 11482981

International Standard Serial Number (ISSN)

  • 1525-0016

Digital Object Identifier (DOI)

  • 10.1006/mthe.2001.0426


  • eng

Conference Location

  • United States