Targeted gene correction: a new strategy for molecular medicine.
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.
Ye
S
S
Kimeragen Inc., Newtown, PA 18940, USA.
Cole-Strauss
A C
AC
Frank
B
B
Kmiec
E B
EB
D000818:Animals; D018390:Gene Targeting; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D006801:Humans; D009841:Oligonucleotides; D010641:Phenotype
D009841:Oligonucleotides