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
-
Stable-Isotope Dilution HPLC-Electrospray Ionization Tandem Mass Spectrometry Method for Quantifying Hydroxyurea in ...Background: Sickle cell anemia (SCA) i...
-
Step toward gene therapy for sickle cell diseaseA team of researchers at the Stanford Un...
-
Ironwood Pharmaceuticals Announces FDA Orphan Drug Designation for Olinciguat for the Treatment of Sickle Cell Disea...Ironwood Pharmaceuticals, Inc., a commer...
-
Hydroxyurea effectiveness in children and adolescents with sickle cell anemia: A large retrospective, population-bas...The clinical efficacy of hydroxyurea in ...
-
Statement from FDA Commissioner on agency’s efforts to advance development of gene therapiesOnce just a theory, gene therapies are...
-
Siklos (hydroxyurea) now available through ProCare Pharmacy Care’s Siklos At Home programMedunik USA announced today that...
-
New Guidelines for Managing Sickle Cell Disease Presented at ACP Internal Medicine MeetingNew guidelines for therapeutic strategie...
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.
