Google
Trusted Resources: News & Events
Latest announcements and gatherings
Step toward gene therapy for sickle cell disease
A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder.
The team went on to demonstrate that the mended cells could make a functioning hemoglobin molecule, which carries oxygen in normal red blood cells, and then successfully transplanted the stem cells into mice. The researchers say the study represents a proof of concept for the repair of blood-borne genetic diseases, such as sickle cell disease and thalassemia.
+myBinderRelated Content
-
Targeted hydroxyurea education after an emergency department visit increases hydroxyurea use in children with sickle...Objective: To evaluate the impact of an...
-
Infographic: Understanding Sickle Cell Disease – ASHhttps://www.onescdvoice.com/wp-content/u...
-
MaxCyte and U.S. National Institutes of Health’s National Heart, Lung, and Blood Institute enter cooperative r...MaxCyte, Inc. today announces it has e...
-
Stable engraftment with haploidentical peripheral blood stem cell transplantation in adult patients with sickle cell...The widespread application of allogeneic...
-
In France, Boy Becomes First Sickle Cell Disease Patient to be Treated With Gene TherapyA 13-year-old boy with sickle cell disea...
-
The Family Perspectivehttp://www.sicklecelltransplantconsortiu...
-
Gene Therapy: Your Questions Answeredhttps://www.youtube.com/watch?v=5ChXI6cS...
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.