Panel C shows naturally occurring variants that cause hereditary persistence of fetal hemoglobin (HPFH). Single-base mutations that lead to increased γ-globin gene expression are indicated.

You might have wondered why some people with sickle cell disease seem to live relatively normal lives while others spend half ...

"Sickle cell disease is a devasting disorder that affects approximately 100,000 people in the U.S., leading to anemia, severe ...

leading to increased production of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimicking the effects of naturally occurring variants seen in hereditary persistence of fetal ...

Editas Medicine's in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to ...

leading to increased production of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimicking the effects of naturally occurring variants seen in hereditary persistence of fetal ...

In all, 35 consecutive patients with transfusion-dependent thalassaemia underwent transplantation with unrelated umbilical cord blood (UCB) units at Chang Gung Children's Hospital between October ...

This clinically validated approach targeting HBG1/2 promoters to upregulate fetal hemoglobin (HbF) is in pre-clinical development as a potential transformative in vivo gene editing medicine for ...

This innovative approach aims to increase fetal hemoglobin levels as a treatment for sickle cell disease and beta thalassemia. In studies with humanized mice and non-human primates, the tLNP ...