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 ...

Medical research

Researchers develop safe way to repair sickle cell disease genes

Researchers at the Salk Institute for Biological Studies have developed a way to use patients' own cells to potentially cure sickle cell disease and many other disorders caused by mutations in a gene that helps produce blood ...

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, ...

Medications

Drug approved for inherited blood disorder

(HealthDay)—Exjade (deferasirox) has been approved by the U.S. Food and Drug Administration to remove excess iron in the blood among people with a genetic blood disorder called non-transfusion-dependent thalassemia (NTDT).

Genetics

Can gene therapy cure fatal diseases in children?

In order for the body to function, a balance is necessary between the cells that build up the bones in our skeletons and the cells that break them down. In the disease malignant infantile osteopetrosis, MIOP, the cells that ...

Health

Sanctions affecting 6 million patients in Iran: report

Some six million patients in Iran are affected by Western economic sanctions as import of medicine is becoming increasingly difficult, a governmental paper reported Sunday quoting a health official.

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