Gene therapy. Principles and potential applications.

Abstract

BACKGROUND: Gene therapy represents an exciting new possibility for the treatment of rare genetic disorders and common multifactorial diseases. OBJECTIVE: To provide an overview of the principals of gene therapy and to outline the current progress of human gene therapy trials. DISCUSSION: Gene therapy is a novel approach to treat, cure or prevent disease by changing the expression of a person's genes. Typically gene therapy involves using a vector such as a virus to deliver a therapeutic gene to the appropriate target cells. Gene therapy is still in its infancy and is not yet available outside clinical trials. Gene therapy was originally envisaged as a treatment of monogenic disorders, but the majority of gene therapy trials now involve the treatment of cancer, infectious diseases and vascular disease. Human gene therapy raises several important ethical issues, in particular the potential use of genetic therapies for genetic enhancement and the potential impact of germline gene therapy on future generations.