Google
Trusted Resources: News & Events
Latest announcements and gatherings
Gene therapy targets sickle-cell disease
Elliott Vichinsky estimates that at least 30% of his adult patients with sickle-cell disease die from preventable causes. Red blood cells are supposed to be shaped like concave discs, but in people with sickle-cell disease, a mutation in a single gene collapses them into a crescent shape. The pointy sickles catch on each other and clog blood vessels. They cut off oxygen to limbs. They cause kidney failure, hypertension, lung problems and strokes — along with bouts of excruciating pain.
These are common and treatable complications, so why the high death rate? Vichinsky attributes it to a lack of infrastructure, such as care centres, to properly monitor adults with sickle-cell disease. This is partly because the disease mainly affects low-income minorities and people in developing countries. “If they were tracked before,” says Vichinsky, “they would not be dead.”
+myBinderRelated Content
-
A Child Prodigy, a Painful Disease, and a Life-Changing Treatmenthttps://www.youtube.com/watch?v=SpzjD1ct...
-
Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approachesSickle cell disease is an inherited diso...
-
SCDAA Statement About Gene Therapy ApprovalOn Dec. 8, 2023, the Food and Drug Admin...
-
The Experience and Health-Related Quality of Life After Haploidentical Stem Cell Transplantation for Adults With Sic...Haploidentical hematopoietic stem cell t...
-
Defeating Sickle Cell Disease with Stem Cells + Gene Therapy: Stem Cells in Your Facehttps://www.youtube.com/watch?v=f3pjyQvB...
-
Sequence Replacement to Cure Sickle Cell DiseaseNext-generation gene editing is already ...
-
FDA grants CRISPR gene therapy fast track designation for sickle cell diseaseThe FDA granted fast track designation t...
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.