The development and regulation of gene repair

Nat Rev Genet. 2003 Sep;4(9):679-89. doi: 10.1038/nrg1156.


A technique that can direct the repair of a genetic mutation in a human chromosome using the DNA repair machinery of the cell is under development. Although this approach is not as mature as other forms of gene therapy and fundamental problems continue to arise, it promises to be the ultimate therapy for many inherited disorders. There is a continuing effort to understand the potential and the limitations of this controversial approach.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Animals
  • DNA Damage
  • DNA Repair*
  • Gene Targeting
  • Genetic Diseases, Inborn / genetics
  • Genetic Diseases, Inborn / therapy
  • Genetic Therapy / adverse effects
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Humans
  • Mutation
  • Plants / genetics
  • Saccharomyces cerevisiae / genetics