Publications [#99811] of Bruce A Sullenger

Papers Published

1. T Watanabe, BA Sullenger, RNA repair: a novel approach to gene therapy., Advanced drug delivery reviews, vol. 44 no. 2-3 (November, 2000), pp. 109-18, ISSN 0169-409X
   (last updated on 2013/05/16)

   Abstract:
   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.

   Keywords:
   Genetic Therapy • Humans • Mutation • Oligonucleotides, Antisense • RNA Editing • RNA Splice Sites • RNA* • RNA, Catalytic • genetics • metabolism • methods*