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Gene-Editing Therapy Helps Nashville Sickle Cell Patients | City Limits

In early December, CRISPR-Cas9 gene editing therapy was approved by the Food and Drug Administration for the treatment of sickle cell in patients ages 12 and up. It’s the first FDA approval for the gene-editing therapy, whose creators won the Nobel Prize in chemistry in 2020. ....

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Gene therapy a potential 'functional cure' for sickle cell disease, beta thalassemia

Treatment with exagamglogene autotemcel conferred clinically meaningful increases in fetal hemoglobin and total hemoglobin levels among patients with beta-thalassemia and sickle cell disease, results of two pivotal studies showed. The latest follow-up data from the CLIMB THAL-111 and CLIMB-SCD-121 trials — presented at American Society for Gene & Cell Therapy Annual Meeting — ....

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