Google
Trusted Resources: Community Center
Online groups, photo galleries and blogs
Sequence Replacement to Cure Sickle Cell Disease
Next-generation gene editing is already transforming the way scientists do research, but it also holds a great deal of promise for the cure of genetic diseases. One of the most tractable genetic diseases for gene editing is sickle cell disease (SCD). The molecular basis has been known since 1949, so it’s relatively well understood. Its root cause is in bone marrow stem cells (aka hematopoietic stem cells, or “HSCs”), which are easy to get to with editing reagents. It’s monogenic and recessive, so you only need to reverse one disease allele for a cure.
+myBinderRelated Content
-
How Is Sickle Cell Disease Treated?Babies with sickle cell disease (SCD) sh...
-
The Opioid Drug Epidemic and Sickle Cell Disease: Guilt by AssociationIn 2008, for the first time in Centers f...
-
Hydroxyurea improves lung function in children with sickle cell diseaseFor the first time, researchers were abl...
-
Is Your Child the Right Candidate for Hydroxyureahttps://www.youtube.com/watch?v=vMvC5rVH...
-
FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe SCD and Transfusion-Dep...BOSTON & ZUG, Switzerland--(BUSINESS...
-
Gene therapy may help cure sickle cell disease, study saysA gene therapy that could provide a perm...
-
Debbie’s Story on BBC2 Hospitalhttps://www.facebook.com/bbctwo/videos/1...
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.
