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Sickle-cell disease is one of the most common genetic disorders, affecting millions of people around the world. It’s caused by a mutation in a gene known as HBB, which makes hemoglobin, a protein that transports oxygen throughout the body. Blood cells with healthy hemoglobin are red and disc-shaped. Cells with abnormal hemoglobin are shaped like sickles used to cut wheat, the characteristic that gives the disease its name.
CRISPR Therapeutics is one of a handful of gene-editing startups pursuing new treatments for sickle-cell. The company’s approach involves isolating stem cells from samples of patients’ blood. Scientists would use CRISPR to activate a genetic switch that would raise the levels of a fetal form of hemoglobin in red blood cells, turning them healthy. This fetal hemoglobin effectively counteracts the effects of the sickle mutation. The modified cells would then be infused back into the patients.
education & researchInitial results from a cohort in a phase 2a study (GBT440-007) evaluating adolescents with sickle cell disease treat...Background: Sickle cell disease (SCD), ...
education & researchPneumococcal vaccination coverage among children with sickle cell anemia, sickle cell trait, and normal hemoglobinBackground: Children with sickle cell a...
news & eventsNew Sickle Cell Disease Research Shows Improved Patient OutcomesEmerging therapies and promising new ins...
news & eventsGene therapy targets sickle-cell diseaseElliott Vichinsky estimates that at leas...
news & eventsSancilio Pharmaceuticals Company, Inc. (SPCI) Receives Rare Pediatric Disease Designation From the US Food and Drug ...Sancilio Pharmaceuticals Company, Inc. (...
videos & visualsASH Clinical Practice Guidelines on Sickle Cell Disease (SCD)https://www.youtube.com/watch?v=tozUcTSN...
news & eventsFighting sickle cell disease by looking back to babyhoodFast-track research focused on developin...
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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.