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
-
Sickle Cell Treatment ‘Life-Changing’ for Brockton BrothersEmmanuel “Manny” Johnson, 21, of Bro...
-
La Jolla Pharmaceutical company announces initiation of pivotal clinical study of LJPC-401 in patients with beta tha...La Jolla Pharmaceutical company today an...
-
CTX001 Earns FDA’s Fast Track Status for Treating Sickle Cell DiseaseThe U.S. Food and Drug Administration (F...
-
The role of nutrition in the pathophysiology and management of sickle cell disease among children: A review of liter...Sickle cell disease (SCD) is one of the ...
-
Sickle Cell Disease Research Shows Progress in Preventing Related Complications and DeathStudies aim to limit pain crises, preven...
-
Red blood cell transfusions during sickle cell anemia vaso-occlusive crises: a report from the magnesium in crisis (...BACKGROUND: Little is known about red b...
-
Gene therapy may help cure sickle cell disease, study saysA gene therapy that could provide a perm...
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.
