Targeted gene repair -- in the arena.
The development of targeted gene repair is under way and, despite some setbacks, shows promise as an alternative form of gene therapy. This approach uses synthetic DNA molecules to activate and direct the cell's inherent DNA repair systems to correct inborn errors. The progress of this technique and its therapeutic potential are discussed in relation to the treatment of genetic diseases.
Kmiec
Eric B
EB
Department of Biology, Delaware Biotechnology Institute, University of Delaware, Newark, Delaware 19716, USA. ekmiec@udel.edu
D000818:Animals; D004260:DNA Repair; D018390:Gene Targeting / Q000379:methods*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans