Home > Encyclopedia of Neurological Disorders > Gene Therapy

Definition

Classic gene therapy is the direct use of genetic material in the treatment of disease. This usually involves inserting a functional gene or DNA fragment into key cells to mitigate, or cure, a disease. A broader definition of gene therapy includes all applications of DNA technology to treat disease. For people with certain neurological conditions such as Parkinson's disease and Canavan disease, initial gene therapy trials have shown promise. Developing gene therapies for treating disorders of the nervous system poses unique challenges, such as how to introduce the therapeutic gene across the blood-brain barrier or how to target the therapeutic gene to one specific area of the brain.

Purpose

Genes play a role in every function...