500,000 children are born with hemoglobinopathies annually, and 80% of them are born in developing countries. According to the Global Prevention and Control of Hemoglobinopathies report, carrier ...

Having just one copy of the sickle cell mutation, known as sickle cell trait, has largely been considered a benign condition.

Researchers at Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine have pioneered a new model that offers a potential platform for developing ...

The evolution of biomedical science can be appreciated through studies of hemoglobin, the oxygen-carrying protein in red cells. Before molecular cloning, the geneticist Arno Motulsky noted, “Many ...

The blood protein hemoglobin and its relative, myoglobin, carry and store life-giving oxygen in many animals. Researchers long thought these complex proteins, with their unique fold, were the only two ...

Mandy Boontanrart, PhD, a postdoc at ETH Zurich in Switzerland, has a particularly personal motivation for her research on a novel genetic therapy for sickle cell disease (SCD). She is a carrier of a ...

Sickle cell disease is characterized by the painful recurrence of vaso-occlusive events. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111; lovotibeglogene autotemcel) consists ...

It seems like a feat of magic. Human DNA, if stretched out into one, long spaghetti-like strand, would measure 2 meters (six feet) long. And yet, all of our DNA is compacted more than 10,000 times to ...

Funding will support clinical development of EPF-001, a first-in-class G9a inhibitor for sickle cell disease and beta thalassemia Company built on pioneering research from RIKEN and Tokyo University ...