Sickle cell disease, which is a group of inherited red blood cell disorders, affects approximately 100,000 people in the U.S., according to the Centers for Disease Control and Prevention.
Sep 10, 2021
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Zolgensma—which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay—is currently the most expensive drug in the world. A one-time treatment of the life-saving drug ...
Sep 1, 2021
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It's been more than six months since Brandy Compton last landed in a hospital emergency room.
Jun 21, 2021
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A team of researchers from the Broad Institute, Harvard University and St. Jude Children's Research Hospital has used a base editing technique in a novel way to treat sickle cell disease (SCD) in mice. In their paper published ...
Researchers at Children's Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production ...
Jun 1, 2021
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A series of laboratory studies is underway in the United States to improve gene therapy worldwide for sickle cell disease, a complex and sometimes deadly heritable blood disorder that dramatically affects the structure and ...
The gene editing technique CRISPR has been making headlines worldwide for its potential to change the genetic makeup of organisms and treat genetic diseases such as sickle cell anemia. It has vast potential in many areas—including ...
Apr 23, 2021
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For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms. Today, ...
Apr 9, 2021
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In 2014, two years after her Nobel Prize-winning invention of CRISPR-Cas9 genome editing, Jennifer Doudna thought the technology was mature enough to tackle a cure for a devastating hereditary disorder, sickle cell disease, ...
Mar 31, 2021
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A single letter difference in a single gene, inherited from both parents, spells a lifetime of anemia and pain for 20 million people, mostly of African ancestry, worldwide. Sickle cell disease (SCD) causes red blood cells ...
Mar 1, 2021
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