RT Book, Section A1 Pyeritz, Reed E. A2 Papadakis, Maxine A. A2 McPhee, Stephen J. A2 Rabow, Michael W. A2 McQuaid, Kenneth R. SR Print(0) ID 1193140345 T1 Evolution of Gene Therapy T2 Current Medical Diagnosis & Treatment 2023 YR 2023 FD 2023 PB McGraw-Hill Education PP New York, NY SN 9781264687343 LK accessmedicine.mhmedical.com/content.aspx?aid=1193140345 RD 2024/03/29 AB The potential for replacing a mutant gene in cells that are most severely affected and thereby causing a disease has long been desired. An alternative is inserting a normal copy of the defective gene in an organ that can produce the defective protein. After considerable research in animals, recent success in humans has shown promise in a few, serious genetic diseases. For example, an adeno-associated virus containing a normal copy of clotting factor IX was inserted into the liver of patients with hemophilia B and largely eliminated the need for intravenous infusions of the missing factor. In a different approach, a normal copy of a mutant gene that causes retinal dystrophy was injected into the retina of a patient resulting in restored vision. Numerous preclinical and clinical trials for other genetic disorders are underway.