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
Pubmed Central ID
- 11072109
International Standard Serial Number (ISSN)
- 0169-409X
Digital Object Identifier (DOI)
- 10.1016/s0169-409x(00)00089-2
Language
- eng
Conference Location
- Netherlands