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A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder.
The team went on to demonstrate that the mended cells could make a functioning hemoglobin molecule, which carries oxygen in normal red blood cells, and then successfully transplanted the stem cells into mice. The researchers say the study represents a proof of concept for the repair of blood-borne genetic diseases, such as sickle cell disease and thalassemia.
education & researchHow I Treat Acute and Persistent Sickle Cell PainSickle pain is the hallmark of sickle ce...
Community CenterHardships, and New Hope, for Sickle Cell PatientsSickle cell disease affects about 100,00...
education & researchPredictors of acute care utilization and acute pain treatment outcomes in adults with sickle cell disease: The role ...Despite its rarity in the United States,...
news & eventsGamida Cell Announces $3.5 Million Grant from the Israeli GovernmentGamida Cell, a leader in cellular and im...
news & eventsSolution to 50-year-old mystery could lead to gene therapy for common blood disordersIn a landmark study that could lead to n...
news & eventsCTX001 Continues to Show Promise in Severe SCDA single dose of CTX001, an experimental...
videos & visualsConstance Benson Cured of Sickle Cell, Speaking at Sickle Cell Disease & Gene Editing Briefing in DChttps://www.youtube.com/watch?v=ROtPSLJl...
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.