Clinical potential of gene therapy: towards meeting the demand

Intern Med J. 2014 Mar;44(3):224-33. doi: 10.1111/imj.12366.

Abstract

Since the discovery that new genetic material could be transferred into human cells resulting in induced expression of genes and proteins, clinicians and scientists have been working to harness the technology for clinical outcomes. This article provides a summary of the current status of developments within the broad discipline of clinical gene therapy. In pursuing the treatment of diverse clinical conditions, a wide variety of therapeutics, each tailor-made, may be required. Gene therapy offers the possibility of accurately and specifically targeting particular genetic abnormalities through gene correction, addition or replacement. It represents a compelling idea that adds a new dimension to our portfolio of credible therapeutic choices.

Keywords: clinical trial; genetic therapy; genetic vector; review.

Publication types

  • Review

MeSH terms

  • Animals
  • Cardiovascular Diseases / genetics
  • Cardiovascular Diseases / therapy
  • Clinical Trials as Topic / methods
  • Clinical Trials as Topic / trends
  • Genetic Therapy / methods*
  • Genetic Therapy / trends*
  • Health Services Needs and Demand / trends*
  • Humans
  • Neoplasms / genetics
  • Neoplasms / therapy
  • X-Linked Combined Immunodeficiency Diseases / genetics
  • X-Linked Combined Immunodeficiency Diseases / therapy