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Sickle Cell Patient Receives CRISPR Gene Therapy
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 & eventsMARAC Advisory Statement: Gene Therapy & Bone Marrow TherapiesPlease note: A previous version of this ...
news & eventsStatement from FDA Commissioner on agency’s efforts to advance development of gene therapiesOnce just a theory, gene therapies are...
news & events1st Patients to Get CRISPR Gene-Editing Treatment Continue to ThriveThe last thing a lot of people want to d...
videos & visualsInvesting in a Cure for Sickle Cell Disease: Lakshmanan Krishnamurti, MDhttps://www.youtube.com/watch?v=Fr5KvM4U...
videos & visualsAdvances in the Treatment of Sickle Cell Disease: L-Glutamine, Crizanlizumab & Gene Therapyhttps://www.youtube.com/watch?v=wkm4axJ3...
education & researchGene Therapy: An Experimental Technique to Treat Genetic DiseasesGene therapy is an experimental techniqu...
news & eventsMaxCyte, Inc. to Present Positive Preclinical Data for Sickle Cell DiseaseMaxCyte, a US-based global company dedic...
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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.