When Dr. Donald Kohn began performing bone marrow transplants in 1987, he saw firsthand the promise and limitations of available treatments for life-threatening blood disorders. That experience planted the seed of a career-defining question: What if you could correct a disease with a patient’s own blood stem cells, potentially curing them for life without a donor?
https://www.uclahealth.org/news/release/what-decade-gene-therapy-research-sickle-cell-disease-taught