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
-
Gamida Cell Announces $3.5 Million Grant from the Israeli GovernmentGamida Cell, a leader in cellular and im...
-
Use of Marijuana in Patients with Sickle Cell Disease Increased the Frequency of Hospitalization for Acute Painful V...Introduction: Recently, there has been g...
-
University Hospitals program uses music to teach about sickle cell (photos, video)The study looked at Seidman's BEATS (Bui...
-
Emmaus, a leader in sickle cell disease treatment, signs agreement with McKesson to expand distribution network for ...Emmaus Life Sciences, Inc., anno...
-
Hydroxyurea: The Best Hope for Sickle Cell Anemia Patientshttps://www.youtube.com/watch?v=L_xSgQWj...
-
Investigational Oral Inhibitor IMR-687 Shows Promising Results in Ongoing Phase 2 Clinical TrialImara’s investigational oral inhibitor...
-
Acceptability of In Utero Hematopoietic Cell Transplantation for Sickle Cell DiseaseBACKGROUND: In utero hematopoietic cell ...
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.