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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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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A Yale-led research team used a new gene editing strategy to correct mutations that cause thalassemia, a form of anemia. Their gene editing technique provided corrections to the mutations and alleviated the disease in mice, ...
Oct 26, 2016
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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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A team of researchers led by scientists at Weill Cornell Medical College has designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also ...
Mar 27, 2012
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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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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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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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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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