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
-
Crizanlizumab 5.0 mg/kg increased the time to first on-treatment sickle cell pain crisis (SCPC) and the likelihood o...Background: In the 52-week SUSTAIN stud...
-
Prophylactic versus selective blood transfusion for sickle cell disease in pregnancyBACKGROUND: Pregnant women with sickle ...
-
New Sickle Cell Disease Research Shows Improved Patient OutcomesEmerging therapies and promising new ins...
-
New Pre-transplant Treatment Regimen Improves Survival of Kids with Sickle Cell Disease, Trial ShowsA new pre-transplant conditioning regime...
-
Prolong Pharma’s Sanguinate Shows Promise in Reverting Shape of Red Blood Cells in SCDProlong Pharmaceuticals’ investigation...
-
Gene therapy may help cure sickle cell disease, study saysA gene therapy that could provide a perm...
-
Phase 1 trial to test under-the-skin injection of sevuparin in sickle cell patientsModus Therapeutics is going to launch ...
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.