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WHAT: A scientist from the National Institutes of Health will present promising, early results from a human clinical trial testing a novel gene replacement therapy in people with severe sickle cell disease. Preliminary findings suggest that the approach has an acceptable level of safety and might help patients consistently produce normal red blood...
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Research Features
More than 100,000 people in the United States and an estimated 20 million worldwide suffer from sickle cell disease, a group of inherited, often life-threatening blood disorders that wreak havoc on the body. Mainly affecting African-Americans, Hispanics and Asians, the disease is caused by a mutation in the gene that makes oxygen-carrying hemoglobi...
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Research Feature
When the NHLBI announced the launch of the Cure Sickle Cell Initiative on September 13, many in the sickle cell disease community responded with enthusiasm. Patients, family members, and advocates took to social media and other channels to say they are ready to do their part, starting with spreading the news about the initiative to their neighbors,...
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News Release
The National Institutes of Health (NIH) today announced the launch of a new initiative to help speed the development of cures for sickle cell disease, a group of inherited blood disorders affecting at least 100,000 people in the United States and 20 million worldwide. The Cure Sickle Cell Initiative will take advantage of the latest genetic discove...
Pain is one of the unfortunate hallmarks of sickle cell disease, often growing in intensity as patients age. It is a leading cause of emergency room visits and hospitalizations among people with the disease, and the frequency of those pain-related hospital admissions is a strong predictor of premature death. Researchers have tried for years to find...
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Research Feature
They ranged in age from 15 to 61—four African American women, all with stories to tell about their struggles with sickle cell disease, all with stories about a common experience that helped them through those struggles: participating in clinical trials. It mattered, said the women, all of whom had joined trials funded by the National Institutes of...
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Research Feature
The notion of altering a person’s genes to cure disease used to be the stuff of science fiction. But gene editing experiments aimed at the genetic disorder that causes sickle cell disease are now making their way from the laboratory to clinical trials. And researchers supported by the National Heart, Lung, and Blood Institute (NHLBI) are hoping tho...