Hemoglobin

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Early CRISPR Trial Shows Promise for Sickle Cell Disease

Researchers have reported encouraging early results from a Phase 1/2 clinical trial of a new CRISPR-based gene-editing therapy for sickle cell disease, offering fresh hope for patients living with the inherited blood disorder.Promising Early OutcomesAccording to the study, treated patients showed sustained production of fetal hemoglobin, a form of hemoglobin that can help prevent red blood cells from becoming rigid…

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FDA Approves First Gene Therapy Offering New Hope for Sickle Cell Disease

The U.S. Food and Drug Administration has approved a landmark gene therapy for sickle cell disease, marking a major advance in the treatment of a painful inherited blood disorder that affects hundreds of thousands of people worldwide.How the therapy worksDeveloped by CRISPR Therapeutics and Vertex Pharmaceuticals, the treatment uses a patient’s own blood stem cells, which are modified outside the body to help…

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