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Many human diseases can be traced back to genetic mutations. One way to cure those diseases is to repair the genetic error. But for many reasons, that is far easier said than done. Progress was made in that effort, however, when the CRISPR/Cas9 gene-editing tool was created and introduced to the biomedical research community in 2013.
By 2017, Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics AG announced that they were engineering a treatment for sickle-cell disease. On July 29, 2019, the therapy was used on a human patient.
The patient was willing to be publicly identified. She is Victoria Gray, a 34-year-old mother of four that has sickle cell disease. She has suffered from bouts of agonizing pain since she was an infant, and has been unable to enjoy many normal parts of life. She received the treatment at Sarah Cannon Research Institute in Nashville, Tennessee.
news & eventsSolution to 50-year-old mystery could lead to gene therapy for common blood disordersIn a landmark study that could lead to n...
Community CenterToday’s Faces of Sickle Cell Disease: Jennelle StephensonJennelle Stephenson was diagnosed with S...
videos & visualsGene Therapy & Clinical Trials Webinarhttps://www.youtube.com/watch?v=yYexIUgH...
news & eventsGene-Edited ‘Supercells’ Make Progress In Fight Against Sickle Cell DiseaseDoctors are reporting the first evidence...
news & eventsBluebird Bio Presents Data From LentiGlobin Gene Therapy TrialBluebird Bio’s investigational gene th...
news & eventsBoston Children’s Hospital receives grant for sickle cell disease researchThe Bill and Melinda Gates Foundation aw...
videos & visualsJennifer Doudna on ethics of gene editinghttps://www.youtube.com/watch?v=8Ijr1ccY...
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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.