Google
Trusted Resources: Community Center
Online groups, photo galleries and blogs
3 Gene Editing Approaches for Sickle Cell Disease
Sickle cell disease (SCD) is a perfect candidate for gene editing. It is perhaps the best understood single-gene condition, due to a substitution of a single DNA base in the gene that encodes the beta subunit of hemoglobin, the protein that carries oxygen in the blood.
Red blood cells, which bend into the sickle shape in the disease, obstructing circulation and causing excruciating pain, descend from hematopoietic (“blood-forming”) stem cells (HSCs) in the bone marrow. So theoretically, a patient’s stem cells can be removed, the mutation replaced in them, and then the fixed cells infused back into the patient, circumventing an immune response.
+myBinderRelated Content
-
Unanticipated consequences identified after implementation of a pediatric emergency department (PED)-based intranasa...Background: The 2014 National Heart Lun...
-
Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial)Blood cell membranes in sickle cell dise...
-
Investigational Oral Inhibitor IMR-687 Shows Promising Results in Ongoing Phase 2 Clinical TrialImara’s investigational oral inhibitor...
-
Bluebird bio acquires Durham, NC, manufacturing site for lentiviral vector productionbluebird bio said today it has acquired ...
-
Manage Your Sickle Cell Disease Symptomshttps://www.onescdvoice.com/wp-content/u...
-
Hydroxyurea: The Best Hope for Sickle Cell Anemia Patientshttps://www.youtube.com/watch?v=L_xSgQWj...
-
Sickle Cell Disease, Hydroxyurea: What You Should Knowhttps://www.onescdvoice.com/wp-content/u...
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.
