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
-
Sancilio Pharmaceuticals Company, Inc. Announces Completion of Enrollment in the SCOT Trial in Pediatric Patients Wi...Sancilio Pharmaceuticals Company, Inc. (...
-
Podcast: How New Gene Therapies for SCD May Impact Women’s FertilityAs we closed 2023, the Food and Drug Ad...
-
Alzheimer’s Treatment Memantine Shows Promise in Treating Sickle Cell DiseaseMemantine, a standard treatment for Alzh...
-
Shared Decision Making for Hydroxyurea Treatment Initiation in Children With Sickle Cell AnemiaClinical trials have demonstrated hydrox...
-
Sickle cell patient with severe anemia rapidly improves with voxelotor, case study showsGlobal Blood Therapeutics (GBT) reported...
-
Sickle Cell Patient Receives CRISPR Gene TherapyMany human diseases can be traced back t...
-
Novel Sickle Cell Drug Causes Radical Results in End of Life PatientsThis week the FDA approved a novel treat...
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.