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
-
Cannabinoid receptor-specific mechanisms to alleviate pain in sickle cell anemia via inhibition of mast cell activ...Sickle cell anemia is a manifestation of...
-
Sickle Cell Anemia Toddlers Benefit from Maximal Hydroxyurea Doses, Study FindsIntensifying treatment with hydroxyurea ...
-
Phase 1 trial to test under-the-skin injection of sevuparin in sickle cell patientsModus Therapeutics is going to launch ...
-
Teen is one of the first ever to get his genes editedhttps://www.youtube.com/watch?v=0xv0CBuj...
-
Initial results from a cohort in a phase 2a study (GBT440-007) evaluating adolescents with sickle cell disease treat...Background: Sickle cell disease (SCD), ...
-
Malik was Cured From Sickle Cell Disease (Teaser)https://www.youtube.com/watch?v=RddQ_qvh...
-
First World Cord Blood Day on Nov. 15 to Highlight Cord Blood Uses, Stem Cell ResearchThe inaugural World Cord Blood Day on No...
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
SomeBodyToTalkTo connects people affected by rare diseases with trusted resources, supportive communities, and opportunities to share their experiences.