Google
Trusted Resources: News & Events
Latest announcements and gatherings
In France, Boy Becomes First Sickle Cell Disease Patient to be Treated With Gene Therapy
A 13-year-old boy with sickle cell disease has become the world’s first patient to be successfully treated with gene therapy. The unidentified teenager remains free from sickle crises and other signs of the disease 15 months after his treatment, which was conducted as part of a Phase 1/2 clinical trial (NCT02151526).
The research team, led by Marina Cavazzana, MD, PhD, a hematology professor at Paris-Descartes University in France, inserted an antisickling β-globin gene, with the help of a viral carrier, into the boy’s blood-producing stem cells.
The report, “Gene Therapy in a Patient with Sickle Cell Disease,” appeared in the New England Journal of Medicine.
+myBinderRelated Content
-
MARAC Advisory Statement: Gene Therapy & Bone Marrow TherapiesPlease note: A previous version of this ...
-
Crizanlizumab lowers pain crises in at-risk sickle cell patients, ad-hoc trial data showNovartis’ investigational th...
-
A multicenter randomized controlled trial of intravenous magnesium for sickle cell pain crisis in childrenMagnesium, a vasodilator, anti-inflammat...
-
FDA Approves Hydroxyurea Tablets for Pediatric Patients With Sickle Cell AnemiaThe US Food and Drug Administration (FDA...
-
Pediatric prescriber pratices of hyrdoxyurea in sickle cell diseaseHydroxyurea (HU) has been shown to be ef...
-
Fighting sickle cell disease by looking back to babyhoodFast-track research focused on developin...
-
Voxelotor can Promote Long-Term Benefits in Teens, Adults With SCD, Latest Clinical Data ShowGlobal Blood Therapeutics’ lead therap...
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.