Gene therapy

Gene therapy treats disease by adding, removing, or changing genetic material in a patient’s cells. It can act in cells inside the body or in cells modified outside it and returned.

Viral vector: A modified virus used to deliver genetic material into cells. Viral vectors carry therapeutic genes into target cells, but immune responses and cargo limits shape their use.
Luxturna: A gene therapy that treats inherited retinal dystrophy caused by mutations in RPE65. It was the first FDA-approved gene therapy for an inherited disease, restoring functional RPE65 in retinal cells.
Gene editing: A set of techniques that make targeted changes to DNA sequences. Gene editing can be a gene therapy strategy, but it is also used in research and other settings.
Insertional mutagenesis: Genetic change caused when inserted DNA disrupts or alters the regulation of a host gene. Integrating vectors can activate cancer-related genes or interrupt normal genes, creating a long-term safety risk.
Stanfield Rogers: An American physician who investigated whether viruses could transfer genes to treat human disease. His early work helped establish the idea of using viral gene transfer therapeutically.
Adeno-associated virus: A small virus engineered as a vector for delivering genetic material to cells. AAV is widely used for in-vivo delivery, with tissue targeting and pre-existing immunity affecting treatment.
Zolgensma: A gene replacement therapy for spinal muscular atrophy caused by SMN1 mutations. It illustrates systemic AAV delivery of a functional gene to address a severe inherited disorder.
Gene replacement therapy: A treatment strategy that supplies a functional copy of a gene to compensate for a faulty one. It is a common gene therapy approach, distinct from directly correcting the original mutation.
Immunogenicity: The capacity of a substance to provoke an immune response. Immune reactions against vectors or therapeutic proteins can limit dosing and damage treated cells.
W. French Anderson: An American physician and researcher associated with early clinical gene therapy development. He helped lead the 1990 clinical trial that modified immune cells in a child with ADA-SCID.

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