Google
Trusted Resources: News & Events
Latest announcements and gatherings
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.
+myBinderRelated Content
-
Jennifer Doudna on ethics of gene editinghttps://www.youtube.com/watch?v=8Ijr1ccY...
-
Barriers and facilitators to research participation among adults, and parents of children with sickle cell disease: ...Patient recruitment into sickle cell dis...
-
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell DiseaseBackground: Sickle cell disease is char...
-
Early clinical trial data show gene therapy reversing sickle cell anemiaAfter over a decade of preclin...
-
MARAC Advisory Statement: Gene Therapy & Bone Marrow TherapiesPlease note: A previous version of this ...
-
Gene therapy may help cure sickle cell disease, study saysA gene therapy that could provide a perm...
-
CRISPR deployed to combat sickle-cell anaemiaA mutation in a single DNA letter causes...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
Support for this site is provided by
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.