Born with a deadly blood disease, Clint and Alissa Finlayson's adopted daughters—Ada, nine, and Lily, 12—are the first patients on the West Coast to receive a new gene therapy offered by UCSF Benioff Children's Hospital ...
Oct 28, 2023
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UCSF Benioff Children's Hospital Oakland is the first hospital in the West to administer a newly approved gene therapy to treat beta thalassemia, a rare, genetic blood disorder that causes severe anemia, with a need for lifelong ...
Aug 31, 2023
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Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia. These two common life-threatening anemias afflict millions of individuals across the globe.
Jun 29, 2023
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In a late-breaking abstract presented at the European Hematology Association (EHA) Congress, a group of researchers that includes Children's Hospital of Philadelphia (CHOP) presented new data on an investigational therapy ...
Jun 14, 2022
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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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University of Illinois Chicago is one of the U.S. sites participating in clinical trials to cure severe red blood congenital diseases such as sickle cell anemia or Thalassemia by safely modifying the DNA of patients' blood ...
Jan 21, 2021
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Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and the University of Massachusetts Medical School have developed a strategy to treat two of the most common inherited blood diseases—sickle ...
Mar 28, 2019
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Doubling the low amount of total body radiation delivered to patients undergoing bone marrow transplants with donor cells that are only "half-matched" increased the rate of engraftment from only about 50 percent to nearly ...
Mar 14, 2019
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Sickle cell disease and the blood disorder beta thalassemia affect more than 180,000 Americans and millions more worldwide. Both diseases can be made milder or even cured by increasing fetal hemoglobin (HbF) levels, but current ...
Jan 11, 2017
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Two studies led by investigators at Weill Cornell Medical College shed light on the molecular biology of three blood disorders, leading to novel strategies to treat these diseases.
Mar 25, 2013
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