Google
Trusted Resources: News & Events
Latest announcements and gatherings
BCL11A-based gene therapy for sickle cell disease passes key preclinical test
A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center report today in the Journal of Clinical Investigation.
The work sets the stage for bringing a decades-old discovery about sickle-cell disease to the bedside. A clinical gene therapy trial, using a virus rendered harmless in the laboratory, is expected to launch in the coming year.
+myBinderRelated Content
-
American Society of Gene & Cell Therapy: Blood DisordersBlood is very important to our body. The...
-
Defeating Sickle Cell Disease with Stem Cells + Gene Therapy: Stem Cells in Your Facehttps://www.youtube.com/watch?v=f3pjyQvB...
-
Patient controlled analgesia for adults with sickle cell disease awaiting admission from the emergency departmentBackground: A treatment algorithm for s...
-
Step toward gene therapy for sickle cell diseaseA team of researchers at the Stanford Un...
-
Siklos (hydroxyurea) now available through ProCare Pharmacy Care’s Siklos At Home programMedunik USA announced today that...
-
Voxelotor can Promote Long-Term Benefits in Teens, Adults With SCD, Latest Clinical Data ShowGlobal Blood Therapeutics’ lead therap...
-
APS Scientific Meeting 2019The American Pain Society will hold its ...
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.