DISCLAIMER
The information and materials accessed through or made available for use on any of our Sites, including, any information about diseases, conditions, treatments, or medicines, are for informational purposes only. The Content is not intended to be and is not a substitute for professional medical advice, diagnosis, or treatment, and your participation on our Sites does not create a healthcare professional-patient relationship. You should consult a doctor or other qualified health care professional regarding any questions you have about your health or before making any decisions related to your health or wellness. Call your doctor or 911 immediately if you think you may have a medical emergency.compose your message
message sent
email sent successfully
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.

Related Content
-
news & eventsExperimental Gene Therapy Reverses Sickle Cell Disease for YearsA study of an investigational gene thera...
-
education & researchThe clinical and biochemical effects of treatment with omega-3 fatty acids in patients with homozygous sickle cell d...Background: Chronic inflammation, coagu...
-
education & researchSteps to Better Health for People with Sickle Cell Disease ToolkitIn partnership with the American Society...
-
videos & visualsSickle cell warrior documentaryhttps://www.youtube.com/watch?v=ZxC4vtN2...
-
news & eventsGlobal Sickle Cell Disease Treatment Market 2019–2023| Evolving Opportunities with ADDMEDICA and Bristol-Myers Squ...The global sickle cell disease treatment...
-
news & eventsKids with sickle cell disease aren’t receiving key vaccines, Michigan study findsA significant proportion of children w...
-
videos & visualsAdvances in the Treatment of Sickle Cell Disease: L-Glutamine, Crizanlizumab & Gene Therapyhttps://www.youtube.com/watch?v=wkm4axJ3...
send a message
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.