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 material into cells. Non-viral approaches include lipid nanoparticles, electroporation, and direct delivery of DNA or RNA. Gene editing technologies, notably CRISPR-Cas systems, enable precise modifications of genomic sequences. Treatments may be given in vivo (directly to the patient) or ex vivo (cells are modified outside the body and then transplanted back).

Therapeutic goals include replacing a faulty gene, silencing a harmful gene, correcting mutations, or adding a new gene with beneficial function. Gene therapies may be designed as single-dose treatments or require repeated administration, and outcomes can include improved symptoms, slowed disease progression, or cure of a disease in some cases.

Applications span inherited disorders, such as certain retinal diseases and spinal muscular atrophy, as well as some cancers and rare metabolic diseases. Several therapies have been approved in various regions and represent milestones in precision medicine; examples include Luxturna, Zolgensma, Libmeldy, Strimvelis, and Hemgenix.

Safety and ethical considerations include potential immune responses, off-target editing, insertional mutagenesis, and long-term risks. Regulators emphasize rigorous clinical testing and long-term follow-up, and researchers continue to explore broader indications and delivery methods.