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Story summary
- UCLA research team led by Dr. Donald Kohn is developing a one-time stem cell gene therapy for alpha thalassemia major, aiming for a cure, building on ADA-SCID where 48 of 50 treated children gained immune function.
- The therapy targets DNA to restore hemoglobin production.
- A California Institute for Regenerative Medicine grant will fund preclinical work and prep for FDA submission.
