• Join Today!

Become a member and connect with:

  • An Active Online Community
  • Articles and Advice on SCD
  • Help Understanding Clinical Trials

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.

https://www.onescdvoice.com/wp-content/uploads/2016/01/gene-therappy.jpg

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.

Close