What is the difference between genome editing and gene therapy?
Gene therapy is a process by which one or more copies of a gene are introduced into a patient’s body with the help of a vehicle. This approach repairs the effect of the malfunctioning gene which is causing a genetically-based disease or condition. In order to have a durable effect, the introduced genes must be expressed for a prolonged period of time (ideally, the entire life of the patient).
Genome editing, by contrast, targets disease-causing genes to correct or remove a defect permanently. Such correction will persist throughout the life span of the transformed cells. Depending on the type of cell involved in the treatment and the lifespan of these cells in the body, genome editing treatments may effectively treat diseases either with one single application or by several repeated applications.