Thanks to groundbreaking in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a growing population of children born with that diagnosis — the severe, inherited blood disorder alpha thalassemia — which requires lifelong specialized care.
But a UCLA research team led by gene therapy pioneer Dr. Donald Kohn is developing a one-time stem cell gene therapy treatment for this condition that’s potentially curative. The study findings are detailed in Cell Reports Medicine.
https://www.uclahealth.org/news/release/ucla-scientists-advance-gene-therapy-deadly-blood-disorder