title: Targeted gene correction: a new strategy for molecular medicine.
abstract: Advances, over the past 20 years, in the genetic manipulation of mammalian cells form the scientific basis of gene therapy. A number of strategies are presently being used to replace or augment a dysfunctional gene with a correct copy of itself. Now, a novel approach to correct the dysfunctional gene in the chromosome is being developed. Data obtained from biochemical, cell-based and animal studies suggest that the era of gene repair is dawning. It is now conceivable that inherited and non-inherited disorders might be treated with a small molecular tool designed to fix the mutation directly. Here, the conceptualization of the technique and its barriers to success are discussed.
authors.affiliation: Kimeragen Inc., Newtown, PA 18940, USA.
authors.forename: S
authors.initials: S
authors.lastname: Ye
authors.forename: A C
authors.initials: AC
authors.lastname: Cole-Strauss
authors.forename: B
authors.initials: B
authors.lastname: Frank
authors.forename: E B
authors.initials: EB
authors.lastname: Kmiec
mesh_terms: D000818:Animals; D018390:Gene Targeting; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D006801:Humans; D009841:Oligonucleotides; D010641:Phenotype
chemical_list: D009841:Oligonucleotides