Google
Trusted Resources: News & Events
Latest announcements and gatherings
Gene therapies could transform the treatment of sickle cell disease
Manny Johnson keeps catching himself rubbing a spot on the upper right side of his chest. Starting when he was a teenager, the 21-year-old from Boston depended on a port implanted there to ease his monthly blood transfusions for sickle cell disease, a genetic disorder that caused a stroke when he was three.
But in November, six months after Johnson became the first patient to receive an experimental therapy aimed at curing his disease, the port that had become part of him — requiring special approval to play sports, used when he was excused from school for a day or two every month for treatments — was removed. Johnson hasn’t needed a transfusion or had any symptoms since May.
“It’s like, wow. I’m fascinated by it,” said Johnson, who added that he almost didn’t know what to make of it when his medical team told him they wouldn’t need to see him for three whole months. “I’m so used to being next to them. It’s like: freedom.”
+myBinderRelated Content
-
Fighting sickle cell disease by looking back to babyhoodFast-track research focused on developin...
-
Step toward gene therapy for sickle cell diseaseA team of researchers at the Stanford Un...
-
Teen is one of the first ever to get his genes editedhttps://www.youtube.com/watch?v=0xv0CBuj...
-
BCL11A-based gene therapy for sickle cell disease passes key preclinical testA precision-engineered gene therapy viru...
-
3 Gene Editing Approaches for Sickle Cell DiseaseSickle cell disease (SCD) is a perfect c...
-
Testing Gene Editing for Sickle Cell Diseasehttps://www.youtube.com/watch?v=RGetNC2B...
-
Jennifer Doudna on ethics of gene editinghttps://www.youtube.com/watch?v=8Ijr1ccY...
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.