Home > Haematology > Gene therapy for beta-thalassemia safe, effective up to 8 years, but concerns emerge

Gene therapy for beta-thalassemia safe, effective up to 8 years, but concerns emerge


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Journal
Nature Medicine
Reuters Health - 04/01/2022 - Lentiviral globin gene therapy with reduced-intensity conditioning (RIC) was safe and effective in beta-thalassemia in a 6-8-year follow-up, although cautious monitoring is still necessary, researchers say. Beta-thalassemias are inherited anemias caused by the absent or insufficient production of the beta chain of hemoglobin. "The overall data from this report and others support the hope that globin disorders can be treated genetically," Dr. Michel Sadelain of Memorial Sloan Kettering Cancer Center in New York City told Reuters Health by email. "We have entered an era where therapeutic efficacy is within reach using a variety of genetic approaches, from integrating vectors to gene editing (e.g., with CRISPR). Their differentiation, and ultimate relevance, will be determined by long-term safety. As this study shows, this assessment takes a long time to accomplish." "Many in the field had anticipated that 'myeloablative conditionin...


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