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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.
education & researchStrategies to build trust and recruit African American and Latino community residents for health research: a cohort ...Background: This study used Community P...
education & researchBarriers and facilitators to research participation among adults, and parents of children with sickle cell disease: ...Patient recruitment into sickle cell dis...
news & eventsSCDAA News Advisory: Partial Hold on Gene Therapy TrialOn December 20, the FDA placed a partial...
videos & visualsUsing CRISPR Cas9 to cure sickle cell diseasehttps://www.youtube.com/watch?v=WdkP-RU5...
Community CenterSickle Cell Disease: Gene-Editing Tools Point to Possible Ultimate CureRecent advances in CRISPR/Cas9 gene-edit...
news & eventsCIRM and NHLBI Collaborating to Fund Cell and Gene Therapies for Sickle Cell DiseaseThe California Institute for Regenerativ...
videos & visualsGene Therapy: Your Questions Answeredhttps://www.youtube.com/watch?v=5ChXI6cS...
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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.