The application of DNA repair vectors to gene therapy.
The nature of DNA, the sequence of the human genome and our increased understanding of the genetic basis of many inherited and acquired disorders have made the possibility of curing diseases a reality. The modulation of a host's genome is now the ultimate goal in the treatment of genetic diseases. Historically, gene therapy recognized two very different approaches: gene replacement or augmentation and gene repair. Gene repair precisely targets and corrects the chromosomal mutation responsible for a genetic and/or acquired disorder. Many recent advances have been made in this area of research.
Kren
Betsy T
BT
Department of Medicine, University of Minnesota Medical School MMC 36, Mayo Building A536, 420 Delaware Street, Minneapolis, MN 55455, USA.
Steer
Clifford J
CJ
D000818:Animals; D025063:Chromosome Disorders / Q000628:therapy; D004247:DNA / Q000235:genetics*; D004260:DNA Repair; D004277:DNA, Single-Stranded / Q000235:genetics*; D018390:Gene Targeting / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D016678:Genome; D006801:Humans; D051379:Mice; D011995:Recombination, Genetic
D004277:DNA, Single-Stranded; D004247:DNA