Google
Trusted Resources: News & Events
Latest announcements and gatherings
NHLBI Stepping Up Efforts to Apprise SCD Patients of Therapies and Trials
Wide interest in a CBS 60 Minutes story about a Florida woman with sickle cell disease (SCD) whose symptoms were eased through gene therapy has prompted the National Heart, Lung, and Blood Institute (NHLBI) to raise awareness of other work the National Institutes of Health (NIH) is doing in this disease.
In the March TV segment, Jannelle Stephenson shared her experience of recovery after receiving genetic treatment as part of an ongoing NIH Phase 1/2 clinical trial (NCT02140554). That study is evaluating the safety and efficacy of the investigational gene therapy LentiGlobin in severe sickle cell anemia.
Developed by Bluebird bio, the therapy is designed to raise patients’ long-term hemoglobin levels. Hemoglobin is the molecule that transports oxygen in red blood cells. Researchers extracted stem cells which have the ability to develop into many different cell types in the body — from people with severe sickle cell anemia and injected them with LentiGlobin BB305 viral vector that carries the modified human beta-A-T87Q globin gene. The treated cells were then delivered back into the patients where they started to produce the hemoglobin (HbAT87Q).
+myBinderRelated Content
-
The first two years of life in sickle cell anemia infants: A results of a comprehensive longitudinal studyInfancy is a critical time during which ...
-
First Sickle Cell Patient Dosed in Phase 2a of IMR-687This morning, Imara Inc. announced that ...
-
In France, Boy Becomes First Sickle Cell Disease Patient to be Treated With Gene TherapyA 13-year-old boy with sickle cell disea...
-
Sickle Cell Groups, Pfizer Work to Bring African-Americans into Clinical TrialsIt’s been nearly half a century since ...
-
FDA Awards UNC Researcher $2M Grant to Study Kidney Disease in Sickle Cell AnemiaThe U.S. Food and Drug Administration (F...
-
Global Sickle Cell Disease Treatment Market 2019–2023| Evolving Opportunities with ADDMEDICA and Bristol-Myers Squ...The global sickle cell disease treatment...
-
Could Gene Therapy Cure Sickle Cell Anemia?Nearly 20 years ago, scientists stunned ...
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.