Google
Trusted Resources: News & Events
Latest announcements and gatherings
These patients had sickle-cell disease. Experimental therapies might have cured them
Scientists have long known what causes sickle-cell disease and its devastating effects: a single mutation in one errant gene. But for decades, there has been only modest progress against an inherited condition that mainly afflicts people of African descent.
With advances in gene therapy, that is quickly changing — so much so that scientists have begun to talk of a cure.
In a half-dozen clinical trials planned or underway, researchers are testing strategies for correcting the problem at the genetic level. Already a handful of the enrolled patients, who have endured an illness that causes excruciating bouts of pain, strokes and early death, no longer show signs of the disease.
Among them is Brandon Williams, 21, who lives with his mother in Chicago. Because of his sickle-cell disease, he had suffered four strokes by age 18. The damage makes it hard for him to speak. His older sister died of the disease.
+myBinderRelated Content
-
Sickle-Cell Patients See Hope in CRISPRSickle-cell disease is one of the most c...
-
1st Patients to Get CRISPR Gene-Editing Treatment Continue to ThriveThe last thing a lot of people want to d...
-
Gene therapy targets sickle-cell diseaseElliott Vichinsky estimates that at leas...
-
Bioverativ and Sangamo announce FDA acceptance of IND application for gene-edited cell therapy BIVV003 to treat sick...Bioverativ Inc., a Sanofi company dedi...
-
Alabama Man Free of Sickle Cell After Gene Therapyhttps://www.youtube.com/watch?v=E9vYON8D...
-
Gene-Editing Treatment Shows Promise for Sickle Cell DiseaseScientists are seeing promising early re...
-
Teen is one of the first ever to get his genes editedhttps://www.youtube.com/watch?v=0xv0CBuj...
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.