Gene therapy is performed by introducing therapeutic genes into a patient's cells through a delivery system known as a vector. Most commonly, this vector is a modified virus, such as an adeno-associated virus (AAV) or lentivirus, which has been engineered to carry the desired gene without causing disease. Once administered, the vector effectively transports the new or corrected gene into target cells—such as liver cells for hemophilia or lung cells for cystic fibrosis. Additionally, gene therapy can employ techniques like direct injection into affected tissues or ex vivo methods, where cells are modified outside the body and subsequently reintroduced into the patient. This innovative approach offers promising pathways to treating various genetic disorders by correcting the underlying genetic abnormalities.
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