Google
Trusted Resources: News & Events
Latest announcements and gatherings
Stem cell gene-editing method may be breakthrough for sickle cell research
Researchers have cured mice with a genetic blood disease by correcting mutated genes in blood cell-producing stem cells. The method offers a new therapeutic approach for treating blood disorders, such as sickle cell disease and thalassemia.
The gene-editing technique can be administered easily into living animals and notably decreases off-target effects (side effects), unlike the CRISPR technique, which displays problems in those regards.
The study, “In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery,” was published in Nature Communications.
+myBinderRelated Content
-
FDA Approves Glutamine Powder for Sickle Cell DiseaseThe U.S. Food and Drug Administration ap...
-
bluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Depende...First patient with severe sickle cell di...
-
A Study of Predictors of Clinical Outcomes and Healthcare Utilization in Children with Sickle Cell Disease Undergoin...Introduction: Current advances in alloge...
-
Transfusion Practices and Strategies for Individuals With Hemoglobin DisordersIntroduction • Describe the optimal h...
-
Sickle Cell Disease Research Shows Progress in Preventing Related Complications and DeathStudies aim to limit pain crises, preven...
-
Crizanlizumab lowers pain crises in at-risk sickle cell patients, ad-hoc trial data showNovartis’ investigational th...
-
MaxCyte, Inc. to Present Positive Preclinical Data for Sickle Cell DiseaseMaxCyte, a US-based global company dedic...
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.