Gene therapy

Gene therapy is an approach to treating potentially fatal or disabling diseases by modifying the expression of an individual's genes toward a therapeutic goal. The premise of gene therapy is based on correcting disease at the DNA level and compensating for the abnormal genes.

Replacement gene therapy replaces a mutated or missing gene, most often a tumor suppressor gene, with a normal copy of that gene which serves to keep cell growth and division under control. The p53 gene, the most common gene mutated in cancer has become a prime target for gene replacement, and has met with some success in inhibiting cell growth, inhibiting angiogenesis (the development of a tumor's blood supply), and inducing apoptosis (cell death).

Knockout gene therapy targets the products of oncogenes (a gene that can induce tumor formation) in an effort to render them inactive and reduce cell growth.