Google
Trusted Resources: News & Events
Latest announcements and gatherings
‘Natural’ Gene Mutation May Offer Way of Treating Sickle Cell Disease, Study Says
Introducing a “natural” genetic mutation into the blood cells of people with sickle cell and like diseases, using CRISPR gene editing, was seen to restore the production of fetal hemoglobin and offer a way to treat these disorders, researchers report.
Their study was published in the journal Blood, in an article titled “KLF1 drives the expression of fetal hemoglobin in British HPFH.”
Sickle cell disease (SCD) patients have a genetic defect that leads them to produce hemoglobin S, or sickle hemoglobin, instead of normal hemoglobin. This impairs the ability of red blood cells to bind to oxygen molecules in the lungs, and consequently to deliver oxygen to tissues throughout the body.
+myBinderRelated Content
-
Hydroxyurea for Sickle Cell Disease TreatmentHydroxyurea is a medicine used to reduce...
-
Today’s Faces of Sickle Cell Disease: Courtney Fitzhugh, M.D.Courtney Fitzhugh first became intereste...
-
2017 April 29 SiNERGe Webinar on Bone Marrow Transplantshttps://www.youtube.com/watch?v=HudIvtS_...
-
Predictive factors of daily opioid use and quality of life in adults with sickle cell diseaseObjectives: In adults with sickle cell ...
-
GBT Receives FDA Breakthrough Therapy Designation for Voxelotor for Treatment of Sickle Cell Disease (SCD)Global Blood Therapeutics, Inc. (GBT) to...
-
Imara’s IMR-687 for SCD Receives FDA Rare Pediatric Disease DesignationThe U.S. Food and Drug Administration (F...
-
Let Your Light Shine: Yoga & MeditationPiedmont Health Services and Sickle 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.