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First Patient to Receive Gene Therapy 'Cure' for Beta-Thalassemia Living Pain-Free

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Rahemeen Nabeel , 12 , has been cured of beta-thalassemia, a genetic-inherited blood disorder.

Zynteglo , a medicine developed at the Children’s Hospital of Philadelphia , uses stem cells to create stem cells that can then be reprogrammed to take over for faulty genetic copies.

Zynelgo, and other similar treatments, use “ Yamanaka factors” to transform normal cells into pluripotent stem cells.