AAV gene therapy

AAV gene therapy

AAV gene therapy uses a harmless virus called adeno-associated virus to deliver small pieces of DNA into human cells. The virus acts like a delivery vehicle: scientists replace its harmful parts with a helpful gene that the body needs. Once inside cells, the new gene can produce a missing protein, correct a defect, or change how cells behave. These viruses are popular because they usually do not cause disease and can provide long-lasting gene activity in many tissues. AAV vectors can target specific cell types by choosing the right viral strain and delivery method. They are already used in approved treatments and in many clinical trials for genetic and acquired diseases. There are limits and risks: the DNA cargo they can carry is small, they can trigger immune reactions, and they may not work equally well in every patient. Researchers work to overcome these limits by engineering better AAVs and by testing safer doses and delivery routes. The eye, liver, and muscle are common targets because they are accessible and respond well to AAV delivery. AAV gene therapy offers the promise of one-time or infrequent treatments that correct underlying problems instead of just masking symptoms.