Google
Trusted Resources: News & Events
Latest announcements and gatherings
Could Gene Therapy Cure Sickle Cell Anemia?
Nearly 20 years ago, scientists stunned the world when they announced they had decoded the genes that make up a human being. They hoped to use that genetic blueprint to advance something called gene therapy which locates and fixes the genes responsible for different diseases.
Now, a clinical trial at the National Institutes of Health is doing exactly that in an attempt to cure sickle cell anemia, a devastating genetic disease that kills hundreds of thousands of people around the world every year.
For the past 15 months we’ve been following the scientists, and patients, who are ushering in a genetic revolution.
In the laboratory, Dr. Tisdale and his collaborators created a gene with the correct spelling. Then, to get that gene into the patient, they used something with a frightening reputation: HIV, the virus that causes AIDS. It turns out HIV is especially good at transferring DNA into cells.
Here’s how it works. The corrected gene, seen here in yellow, is inserted into the HIV virus. Then, bone marrow stem cells are taken from of a patient with sickle cell anemia. In the laboratory those cells are combined with the virus carrying that new DNA.
+myBinderRelated Content
-
These patients had sickle-cell disease. Experimental therapies might have cured themScientists have long known what causes s...
-
Sickle Cell Disease: Gene-Editing Tools Point to Possible Ultimate CureRecent advances in CRISPR/Cas9 gene-edit...
-
Testing Gene Editing for Sickle Cell Diseasehttps://www.youtube.com/watch?v=RGetNC2B...
-
In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic DisorderFor the first time, doctors in the U.S. ...
-
MaxCyte, Inc. to Present Positive Preclinical Data for Sickle Cell DiseaseMaxCyte, a US-based global company dedic...
-
SCDAA Gene Therapy Town Hall – Webinarhttps://www.youtube.com/watch?v=J77Gq9yI...
-
bluebird bio Presents New Data from HGB-205 Study of LentiGlobinTM Drug Product in Patients with Transfusion-Depende...First patient with severe sickle cell di...
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.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.