A major challenge in human genetics is understanding which parts of the genome drive specific traits or contribute to disease risk. This challenge is even greater for genetic variants found in the 98% of the genome that does ...
May 4, 2023
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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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Researchers at Children's Hospital of Philadelphia (CHOP) have discovered a new part of the regulatory cascade responsible for silencing fetal hemoglobin in adult cells. The findings, published today in Nature Genetics, refine ...
Aug 8, 2022
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This week, scientists gathered in Washington, DC for the International Summit on Human Gene Editing to discuss a technology called CRISPR-CAS9, which can insert, remove and change the DNA of basically any organism. It is ...
Dec 4, 2015
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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 ...
A gene therapy that could provide a permanent cure for sickle cell disease continues to show success through a third wave of patients, researchers report.
Dec 13, 2021
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Gene therapy trials for sickle cell disease have been showing great promise, even offering hope of a cure. But in early 2021, the trials ground to a halt after reports of blood malignancies in two people in a trial sponsored ...
Feb 15, 2022
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(HealthDay)—Though heart problems or heatstroke generally are to blame for a young athlete's sudden death, experts now know that carrying an aberration called the sickle cell trait also poses substantial risk.
Dec 28, 2012
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University of Wisconsin-Madison engineers have developed methods to observe gene editing in action, and they're putting those capabilities to work to improve genetic engineering techniques.
Jan 24, 2017
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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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