gene therapy

Syllable breakdown

Dictionary meaning

a medical approach that introduces, alters, or replaces genetic material within a person's cells to treat or prevent disease, either by correcting a faulty gene, supplying a functional copy of a missing gene, or introducing a new gene that gives cells a therapeutic property; delivery strategies include viral vectors — especially adeno-associated viruses (AAV) and lentiviruses — and non-viral approaches such as lipid nanoparticles; approved gene therapies include treatments for inherited blindness, spinal muscular atrophy, and haemophilia; the field also encompasses ex vivo approaches in which patient cells are removed, genetically modified, and reinfused, as in CAR-T cancer immunotherapy; challenges include immune responses to vectors, off-target effects, and the high cost of manufacturing