Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease. ...
Dec 21, 2022
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72
UCLA scientists and colleagues have created a first-of-its-kind roadmap that traces each step in the development of blood stem cells in the human embryo, providing scientists with a blueprint for producing fully functional ...
Apr 13, 2022
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220
Scientists have found a way to lower stroke risk in sickle cell patients that opens a gateway of potential treatments for inflammation and clotting stemming from conditions such as COVID-19. For the first time, they investigated ...
Jan 28, 2021
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Cardiovascular medicine, hematology and pulmonary medicine may soon have the first-ever therapies to correct poor tissue oxygenation, a key driver of disease in millions, including peripheral artery disease, sickle cell disease, ...
Mar 1, 2023
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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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A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children's ...
Sep 6, 2016
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Researchers from Dana-Farber/Boston Children's Cancer and Blood Disorders Center have found that changes to a small stretch of DNA may circumvent the genetic defect behind sickle cell disease (SCD). The discovery, published ...
Sep 16, 2015
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A collaborative team of researchers, including Stephan Grupp, MD, Ph.D., Cell Therapy and Transplant Section Chief and Medical Director of the Cell and Gene Therapy Laboratory at Children's Hospital of Philadelphia (CHOP) ...
Aug 10, 2021
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274
A new study suggests the number of deaths due to sickle cell disease is 11 times higher than what is indicated from mortality data sources alone. Sickle cell disease is not just underdiagnosed, but it also increases risk ...
Jun 27, 2023
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59
One of the lingering mysteries of the COVID-19 pandemic is why some people get infected without getting sick and others don't get infected at all, despite exposure.
Apr 4, 2022
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