Google
Trusted Resources: Community Center
Online groups, photo galleries and blogs
Successful Gene Therapy for Sickle Cell Disease
Prior to this report, the only curative therapy for sickle-cell disease was allogeneic bone marrow transplant. But few patients have matching donors, and even if a match is found, lifelong immunosuppression is necessary to prevent rejection. Gene therapy is the future. Here’s how it was done.
First, the researchers harvested stem cells from the child’s bone marrow. These stem cells were exposed to a lentivirus vector containing an alternate hemoglobin gene. Now, here is where it gets really clever. You’d expect that they just put the normal hemoglobin A gene in there, but they didn’t. Instead, they inserted a variant of hemoglobin A with a glutamine-threonine amino acid substitution. This variant has anti-sickling properties. In fact, the variant was discovered in some kids who were expected to have sickle-cell disease but never manifested symptoms.
This is fighting fire with fire. Use a mutated hemoglobin A gene to combat a mutated hemoglobin A gene.
+myBinderRelated Content
-
Prolong Pharma’s Sanguinate Shows Promise in Reverting Shape of Red Blood Cells in SCDProlong Pharmaceuticals’ investigation...
-
Sickle Cell Disease Management & Complications: Sophie Lanzkron MD of Johns HopkinsStuck on sickle cell disease? We hammer ...
-
Sickle Cell Disease and Marrow/Stem Cell Donationhttps://www.youtube.com/watch?v=mstThQc7...
-
A randomized controlled pilot study feasibility of a tablet-based guided audio-visual relaxation intervention for re...AIM: To test feasibility of a guided aud...
-
Gene therapy may help cure sickle cell disease, study saysA gene therapy that could provide a perm...
-
Determining Adherence to Quality Indicators in Sickle Cell Anemia Using Multiple Data SourcesINTRODUCTION: Advances in primary prophy...
-
New treatment plan leads to better pain control for acute sickle cell crisisThere’s new hope for the 70,000 to 80,...
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.
