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
-
Safety and early hints of benefit seen in phase 1b trial of PF-04447943Sickle cell disease (SCD) patients were ...
-
Hydroxycarbamide for Children With Sickle Cell Disease (SCD)Hydroxycarbamide is a medicine taken by ...
-
Sickle cell disease: Challenges and progressDespite a long history of knowing the ge...
-
SCDAA News Advisory: SCDAA Statement on Exa-cel Gene TherapyOn October 31, 2023, the Food and Drug A...
-
Sickle Cell Patient Receives CRISPR Gene TherapyMany human diseases can be traced back t...
-
Sisterly bond behind sickle-cell curehttps://www.youtube.com/watch?v=9S-xVGsR...
-
Review Board Finds Global Blood’s Sickle Cell Disease Therapy Voxelotor Is SafeAn independent review board has found Gl...
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.
