Pediatrics

Pioneering Gene Therapy by UCSF Benioff Children's Hospital Oakland Researchers Demonstrates Therapy Can Eliminate Life-Long Need for Transfusions in Patients with Incurable Blood Disorder

Interim results of clinical trials by investigators at UCSF Benioff Children’s Hospital Oakland and Ann and Robert H. Lurie Children’s Hospital of Chicago reveal that a majority of the 22 patients in two Phase 1/2 studies followed for two years or longer remained free from transfusions.

The results of the trials “Gene Therapy in Patients with Transfusion-Dependent╬▓-Thalassemia,” are published today in the New England Journal of Medicine (NEJM). The results are from two separate, two-year clinical studies using LentiGlobin(r) gene therapy to stop or reduce chronic blood transfusions in patients with transfusion-dependent ╬▓-thalassemia (TDT).

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