Targeted gene repair

Targeted gene repair is a technique used to correct a mutation at a specific site in an episome or chromosome. It uses synthetic oligonucleotides together with the cell's inherent DNA repair system to direct single base pair changes.

Latest Research and Reviews

News and Comment

  • News and Views |

    Counteracting splice defects in the CEP290 gene using RNA antisense oligonucleotides or Cas9-mediated gene editing is a therapeutic strategy for Leber congenital amaurosis type 10—a severe untreatable retinal dystrophy leading to childhood blindness.

    • José Alain Sahel
    •  & Deniz Dalkara
    Nature Medicine 25, 198-199
  • Research Highlights |

    Cas9 induces larger-than-anticipated mutations in mouse and human cells. In the latter, efficient editing depends on inhibition of the DNA-damage-repair protein p53.

    • Nicole Rusk
    Nature Methods 15, 569
  • Editorial |

    With the advent of precision genome editing, the ability to modify living organisms has proceeded with remarkable speed and breadth. Any application of this technology to the human germ line must be tightly coupled to deliberate consideration of the consequences, both scientific and social, of introducing heritable alterations to the human population. We recommend constant oversight and evaluation of human germline genome editing to balance prudence with discovery, and risk with progress.

    Nature Genetics 49, 653