Google
Trusted Resources: News & Events
Latest announcements and gatherings
Sickle-Cell Patients See Hope in CRISPR
Sickle-cell disease is one of the most common genetic disorders, affecting millions of people around the world. It’s caused by a mutation in a gene known as HBB, which makes hemoglobin, a protein that transports oxygen throughout the body. Blood cells with healthy hemoglobin are red and disc-shaped. Cells with abnormal hemoglobin are shaped like sickles used to cut wheat, the characteristic that gives the disease its name.
CRISPR Therapeutics is one of a handful of gene-editing startups pursuing new treatments for sickle-cell. The company’s approach involves isolating stem cells from samples of patients’ blood. Scientists would use CRISPR to activate a genetic switch that would raise the levels of a fetal form of hemoglobin in red blood cells, turning them healthy. This fetal hemoglobin effectively counteracts the effects of the sickle mutation. The modified cells would then be infused back into the patients.
+myBinderRelated Content
-
Prevention of acute chest syndrome by implementing a standardized process to improve incentive spirometry use in hos...Acute chest syndrome (ACS) is the second...
-
Hardships, and New Hope, for Sickle Cell PatientsSickle cell disease affects about 100,00...
-
Bioverativ and Sangamo announce FDA acceptance of IND application for gene-edited cell therapy BIVV003 to treat sick...Bioverativ Inc., a Sanofi company dedi...
-
Kids with sickle cell disease aren’t receiving key vaccines, Michigan study findsA significant proportion of children w...
-
Transcutaneous electrical nerve stimulation (TENS) for pain management in sickle cell diseaseAs defined by the American Physical Ther...
-
Emmaus Life Sciences, Inc. to present results of phase 3 study of endariTM (L-glutamine oral powder) at 59th America...Emmaus will present results of a Phase 3...
-
What’s Inside My Medicine Cabinet?Living with sickle cell disease, I rely ...
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
SomeBodyToTalkTo connects people affected by rare diseases with trusted resources, supportive communities, and opportunities to share their experiences.