Gene Delivery Systems and Gene Therapy

Abstract: Gene therapy involves introducing genetic material (transgenes) into specific cells to correct genetic defects or introduce new functions, aiming for therapeutic effects at the cellular level. This approach modifies the human genome to treat or prevent diseases by repairing mutated genes or implementing precise genetic alterations. Gene delivery systems are methods used to transfer, remove, or modify DNA or RNA in cells to address medical conditions. They are classified into viral vectors, which offer high efficiency but carry risks such as immune reactions and unintended mutations, and non-viral vectors, which are safer but typically less efficient. Gene therapy represents a cutting-edge strategy for treating or preventing illnesses, though it remains largely experimental. Its applications are being explored in genetic disorders, cancer, and viral infections like AIDS. Two main types exist: germline therapy, which is heritable but controversial, and somatic therapy, which is non-heritable and generally safer. Despite ongoing advancements, challenges related to delivery efficiency, safety, and ethical considerations persist, necessitating continued research to enhance clinical outcomes.