β-Thalassemia

JOSHA’S CRITICAL REVIEW OF “GENE THERAPY IN PATIENTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA” BY A.A. Thompson et al. N Engl J Med 2018; 378:1479- 1493.

THESE INTERIM RESULTS OF TWO PHASE I/II TRIALS ARE REMARKABLE. THOMPSON AND COLLEAGUES DEMONSTRATE THE PROGRESS OF CLINICAL GENE THERAPY FOR MONOGENETIC DISEASES WITH A MEDIAN OBSERVATION TIME OF 26…