Diseases, Conditions, Syndromes

Understanding sickle cell disease

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.

Medical research

Positive preliminary data on CRISPR treatment for blood diseases

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) ...

Diseases, Conditions, Syndromes

CRISPR technology to cure sickle cell disease

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 ...

Medical research

Researchers optimize gene editing for SCD and beta thalassemia

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 ...

Oncology & Cancer

Blood diseases cured with bone marrow transplant

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 ...

Obstetrics & gynaecology

Baby born in world's first in utero stem cell transplant trial

UC San Francisco researchers have safely transplanted a woman's stem cells into her growing fetus, leading to the live birth of an infant with a normally fatal fetal condition. The infant, who had been critically ill during ...

Medical research

Fighting sickle cell disease using a medication for type 2 diabetes

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 ...

Genetics

Scientists edit gene mutations in inherited form of anemia

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, ...

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