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Abstract

Gene therapy, a groundbreaking approach in biomedical sciences, promises to treat and potentially cure genetic disorders by altering defective genes. This article explores its mechanisms, recent advancements, and the challenges impeding its widespread adoption, while forecasting its future in medicine. Through a review of clinical trials and research, it examines how gene therapy tackles diseases like cystic fibrosis, muscular dystrophy, and cancer. Findings highlight significant successes—such as restored vision in inherited blindness—alongside hurdles like delivery inefficiencies, immune responses, and ethical dilemmas. The study underscores gene therapy’s revolutionary potential, tempered by obstacles, and envisions a future where precision and accessibility redefine healthcare.