Journal of Orofacial Sciences (Jan 2013)

Gene therapy: An overview

  • Sudip Indu,
  • Venkatapathy Ramesh,
  • Nirima Oza,
  • Karthikshree V Prashad

DOI
https://doi.org/10.4103/0975-8844.124249
Journal volume & issue
Vol. 5, no. 2
pp. 83 – 87

Abstract

Read online

Gene therapy "the use of genes as medicine" involves the transfer of a therapeutic or working copy of a gene into specific cells of an individual in order to repair a faulty gene copy. The technique may be used to replace a faulty gene, or to introduce a new gene whose function is to cure or to favorably modify the clinical course of a condition. The objective of gene therapy is to introduce new genetic material into target cells while causing no damage to the surrounding healthy cells and tissues, hence the treatment related morbidity is decreased. The delivery system includes a vector that delivers a therapeutic gene into the patient′s target cell. Functional proteins are created from the therapeutic gene causing the cell to return to a normal stage. The vectors used in gene therapy can be viral and non-viral. Gene therapy, an emerging field of biomedicine, is still at infancy and much research remains to be done before this approach to the treatment of condition will realize its full potential.

Keywords