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
-
Orphan Drugs for Sickle Vaso-occlusion: Dawn of a New Era of Targeted TreatmentWhile an orphan disease in the USA, sick...
-
SCD Patients Benefit From Early Rivipansel Treatment for VOCs, New Analyses ShowStarting treatment with rivipansel (GMI-...
-
Coexistent Sickle Cell Disease Has No Impact on the Safety or Outcome of Lytic Therapy in Acute Ischemic Stroke: Fin...BACKGROUND AND PURPOSE: The recommended ...
-
Pediatric sickle cell study stopped early due to positive resultsA national sickle cell disease study inv...
-
First systemic evidence for safety of tPA in stroke patients with sickle cell diseaseIn the largest report on acute stroke in...
-
Could Gene Therapy Cure Sickle Cell Anemia?Nearly 20 years ago, scientists stunned ...
-
Could Gene Therapy Cure Sickle Cell Anemia?https://www.cbsnews.com/video/could-gene...
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.
