Gene therapy (ADA-SCID, Luxturna, Zolgensma)
Terapia génica (ADA-SCID, Luxturna, Zolgensma)
- Date
- 1990-2017 · confidence high
- Place
- NIH, Bethesda, Maryland, United States (1990); Children's Hospital of Philadelphia and Spark Therapeutics (Luxturna, 2017)
- Credited to
- William French Anderson, Michael Blaese and Kenneth Culver (1990); Jean Bennett and Katherine High (Luxturna); Jesse Gelsinger (death in 1999)
- People
- 👤 Jean Bennett1954–👤 Jesse Gelsinger1981–1999👤 Katherine A. High👤 William French Anderson1936–
- Field
- Life & health
What it is
Introduction of a functional gene into a patient's cells using viral vectors to cure hereditary diseases. After decades of setbacks, Luxturna (2017) was the first approved in the United States.
What it led to
- One-time cures and price tags of millions of dollars per dose
- Viral vectors and in vivo gene editing
- Regulation of advanced therapy
Built on
- Recombinant DNA genetic engineering1973
- retroviral and AAV vectors
- bone marrow transplant