Gene Therapy: An Overview of Approaches and Issues

Abstract

For over 25 years, scientists have been researching and testing gene therapy techniques, but this work has only resulted in a single FDA-approved therapy: Kymriah™ from Novartis. Kymriah™ was approved in the U.S. in August 2017, and genetically modifies a patient’s own immune cells to seek out and destroy abnormal blood cells. Although clinical trials continue to test and refine different gene therapy approaches, understanding and evaluating the risks associated with treatment may be overwhelming to patients and caregivers alike. This article attempts to provide readers with an introduction to gene therapy so that anyone considering treatment or caring for someone that is being treated can better understand how gene therapies works, and some of the issues we may have to deal with before gene therapy can become more common.