Genetherapy
Gene therapy is a medical field that uses genetic material to treat or prevent disease. It aims to correct defective genes, compensate for abnormal genes, or introduce new genes that help the body fight disease or restore function. Therapies can be delivered to specific tissues or to entire organs using various strategies.
Delivery systems include viral vectors, such as adeno-associated virus (AAV), adenovirus, and lentivirus, which transport genetic
Therapeutic goals include replacing a faulty gene, silencing a harmful gene, correcting mutations, or adding a
Applications span inherited disorders, such as certain retinal diseases and spinal muscular atrophy, as well as
Safety and ethical considerations include potential immune responses, off-target editing, insertional mutagenesis, and long-term risks. Regulators