RNA repair: a novel approach to gene therapy.

Journal Article (Journal Article;Review)

Treatment of genetic disorders by gene therapy has conventionally been attempted through the transfer of a wild type version of a gene to the cells of a patient harboring defective copies of a disease associated gene. Despite significant advances using this paradigm, several technical hurdles must still be overcome before this 'gene replacement' approach will become useful in the treatment of a variety of genetic maladies. Such limitations have led a number of researchers to begin to investigate alternative strategies to genetic therapy. Repair of mutant genetic instructions represents a fundamentally different approach to genetic therapy that may have significant advantages over gene replacement. Herein, we will discuss recent advances using repair of mutant RNAs as a novel means to correct genetic deficiencies.

Full Text

Duke Authors

Cited Authors

  • Watanabe, T; Sullenger, BA

Published Date

  • November 15, 2000

Published In

Volume / Issue

  • 44 / 2-3

Start / End Page

  • 109 - 118

PubMed ID

  • 11072109

International Standard Serial Number (ISSN)

  • 0169-409X

Digital Object Identifier (DOI)

  • 10.1016/s0169-409x(00)00089-2


  • eng

Conference Location

  • Netherlands