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Introducing a “natural” genetic mutation into the blood cells of people with sickle cell and like diseases, using CRISPR gene editing, was seen to restore the production of fetal hemoglobin and offer a way to treat these disorders, researchers report.
Their study was published in the journal Blood, in an article titled “KLF1 drives the expression of fetal hemoglobin in British HPFH.”
Sickle cell disease (SCD) patients have a genetic defect that leads them to produce hemoglobin S, or sickle hemoglobin, instead of normal hemoglobin. This impairs the ability of red blood cells to bind to oxygen molecules in the lungs, and consequently to deliver oxygen to tissues throughout the body.
education & researchThe influence of the age of adults with sickle cell disease on the uptake, utilization and efficacy of HydroxyureaDespite the efficacy of Hydroxyurea (HU)...
news & eventsMatthew Porteus awarded grant for sickle cell anemia workThe California Institute for Regenerativ...
education & researchAlloimmunization in sickle cell disease: changing antibody specificities and association with chronic pain and decre...BACKGROUND: Alloimmunization remains a ...
news & eventsImara Reports Favorable Preclinical and Phase 1 Data on IMR-687 in Sickle Cell DiseaseImara Inc. today announced it will repor...
videos & visualsHydroxyurea Treatment for Children with Sickle Cell Diseasehttps://www.youtube.com/watch?v=nx7MvMje...
Community CenterToday’s Faces of Sickle Cell Disease: John Tisdale, MDFor many years, John Tisdale has been wo...
news & eventsSickle Cell Disease Association of America, Inc. and Hemanext® Inc. Form New Strategic PartnershipSickle Cell Disease Association of Ameri...
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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.