Google
Trusted Resources: News & Events
Latest announcements and gatherings
Experimental Gene Therapy Reverses Sickle Cell Disease for Years
A study of an investigational gene therapy for sickle cell disease has found that a single dose restored blood cells to their normal shape and eliminated the most serious complication of the disease for at least three years in some patients.
Four patients at Columbia University Irving Medical Center/NewYork-Presbyterian participated in the multicenter study, the first to report on such long-term outcomes of a sickle cell gene therapy. The study was published online Dec. 12 in the New England Journal of Medicine with John F. Tisdale, MD, senior investigator at the NIH’s National Heart, Lung and Blood Institute, as corresponding author.
The single-dose therapy, tested on 35 adults and adolescents with sickle cell disease, essentially corrected the shape of the patient’s red blood cells, but also completely eliminated episodes of severe pain, caused when rigid, crescent-shaped red blood cells clump together and block blood vessels. The painful episodes often result in widespread organ damage. Such episodes are a frequent cause of emergency department visits and hospitalizations and lead to early death.
+myBinderRelated Content
-
Bioverativ and Sangamo announce FDA acceptance of IND application for gene-edited cell therapy BIVV003 to treat sick...Bioverativ Inc., a Sanofi company dedi...
-
Phase 3 study of L-Glutamine in sickle cell disease: Analyses of time to first and second crisis and average cumulat...A Phase 3, double-blind, randomized, pla...
-
Review Board Finds Global Blood’s Sickle Cell Disease Therapy Voxelotor Is SafeAn independent review board has found Gl...
-
A Phase 3 Randomized Trial of Voxelotor in Sickle Cell DiseaseBackground: Deoxygenated sickle hemog...
-
MaxCyte, Inc. to Present Positive Preclinical Data for Sickle Cell DiseaseMaxCyte, a US-based global company dedic...
-
ATS Releases Guidelines for Home Oxygen Therapy in Children With Sickle Cell DiseaseThe American Thoracic Society (ATS) rele...
-
Matthew Porteus awarded grant for sickle cell anemia workThe California Institute for Regenerativ...
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.