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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A clinical trial of a new gene therapy for sickle cell disease that has shown promise has been halted after two participants were diagnosed with cancer.
Feb 24, 2021
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Researchers at Children's Hospital of Philadelphia (CHOP) have developed a gene therapy vector for blood disorders like sickle cell disease and beta-thalassemia that is potentially safer and more effective than those currently ...
Jan 29, 2021
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Scientists are seeing promising early results from the first studies testing gene editing for painful, inherited blood disorders that plague millions worldwide, especially Black people.
Dec 5, 2020
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For Evie Junior, living with sickle cell disease has been like running a marathon.
Nov 30, 2020
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Sickle cell trait and sickle cell disease are risk factors for experiencing kidney function decline among Black individuals. A new study indicates that the risk of acute kidney injury is also higher in adults with sickle ...
Oct 24, 2020
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It's still too soon to try to make genetically edited babies because the science isn't advanced enough to ensure safety, says an international panel of experts who also mapped a pathway for any countries that want to consider ...
Sep 3, 2020
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A pilot gene therapy treatment for sickle cell disease, restoring patients' ability to make fetal hemoglobin, has produced good results in the first three patients to receive it. Investigators at Boston Children's Hospital ...
Jan 10, 2020
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Thanks to the 'cut and paste' gene editing technique CRISPR, scientists are homing in on a cure for sickle cell disease—a genetic blood disorder—while other research is looking at how to expand the potential of CRISPR-based ...
Dec 16, 2019
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In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering—by editing her genome.
Dec 5, 2019
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