Gene therapy is a medical approach that aims to treat, prevent, or cure disease by introducing, modifying, or replacing genetic material — DNA or RNA — within a patient's cells to restore normal gene function, silence a pathogenic gene, or introduce a new therapeutic gene. It can be delivered in vivo (directly into the patient's body) or ex vivo (by modifying cells outside the body before reinfusion), using viral vectors — such as AAV, adenoviral vectors, or lentiviral vectors — or non-viral delivery systems.
