Google
Trusted Resources: News & Events
Latest announcements and gatherings
Matthew Porteus awarded grant for sickle cell anemia work
The California Institute for Regenerative Medicine has awarded MATTHEW PORTEUS, associate professor of pediatrics, a grant of $5.2 million to lay the groundwork for a clinical trial of a possible treatment for sickle cell disease.
Porteus has shown that he can take human blood stem cells with the gene defect that causes sickle cell disease and use gene-editing tools to repair the faulty gene. He also showed that he could successfully transplant those repaired blood stem cells into mice.
“We are extremely excited that, with CIRM support, we may be able to use gene correction to treat this terrible disease,” Porteus said.
+myBinderRelated Content
-
Fetal Haemoglobin in Sickle-cell Disease: From Genetic Epidemiology to New Therapeutic StrategiesSickle-cell disease affects millions of ...
-
Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approachesSickle cell disease is an inherited diso...
-
Defeating Sickle Cell Disease with Stem Cells + Gene Therapy: Stem Cells in Your Facehttps://www.youtube.com/watch?v=f3pjyQvB...
-
CTX001 Earns FDA’s Fast Track Status for Treating Sickle Cell DiseaseThe U.S. Food and Drug Administration (F...
-
Acetylon Presents Preclinical Data Demonstrating the Utility of Selective HDAC1,2 Inhibition by ACY-957 to Induce Ga...Acetylon Pharmaceuticals, Inc., the lead...
-
Nashville Doctor Performs First Successful Gene Editing Procedure on Sickle Cell Anemia Patienthttps://www.youtube.com/watch?v=s7TPRomV...
-
Debbie’s Story on BBC2 Hospitalhttps://www.facebook.com/bbctwo/videos/1...
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.