Google
Trusted Resources: Community Center
Online groups, photo galleries and blogs
Sickle Cell Disease: Gene-Editing Tools Point to Possible Ultimate Cure
Recent advances in CRISPR/Cas9 gene-editing tools, which the blog has highlighted in the past, have renewed hope that it might be possible to cure sickle cell disease by correcting DNA typos in just the right set of cells. Now, in a study published in Science Translational Medicine, an NIH-funded research team has taken an encouraging step toward this goal. For the first time, the scientists showed that it’s possible to correct the hemoglobin mutation in blood-forming human stem cells, taken directly from donors, at a frequency that might be sufficient to help patients.
+myBinderRelated Content
-
Hydroxyurea Therapy Prevents High Risk Trans-Cranial Doppler Development in Children with Sickle Cell DisordersAnnual evaluation by Trans-Cranial Doppl...
-
Today’s Faces of Sickle Cell Disease: John Tisdale, MDFor many years, John Tisdale has been wo...
-
Sickle Cell Disease Tips for Healthy LivingCommon illnesses, like the flu, can quic...
-
Ghana Launches Partnership With Novartis to Improve Diagnosis and Treatment of People With Sickle Cell DiseaseThe Government of Ghana announced a new ...
-
Phase 1 trial to test under-the-skin injection of sevuparin in sickle cell patientsModus Therapeutics is going to launch ...
-
A new Sickle Cell Disease Drug Holds Much Promise but Most Sufferers Won’t be Able to Afford itThe regulatory approval of a groundbreak...
-
Phase 3 Trial Recruiting to Test Rivipansel for Vaso-Occlusive Crisis in SCDGlycoMimetics, in collaboration with Pfi...
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.
