There are many types of sickle cell disease. The majority of cases are divided into three subtypes depending on which genes you inherited: Hemoglobin is an iron-containing protein found in your ...
"Sickle cell disease has a broad spectrum of severity ... instructions for his red blood cells to make a fetal form of hemoglobin. (Unlike "adult" hemoglobin, fetal hemoglobin doesn't cause ...
The new gene therapies for sickle cell disease—including the gene-editing ... that raises fetal hemoglobin levels. The FDA approved it in 1998. But Orkin says hydroxyurea and other available ...
They didn’t know that she carried a genetic blueprint for a misshapen molecule; that her hemoglobin proteins ... newborn’s heel and test it for sickle cell disease. Catch it early and you ...
In sickle cell disease, a gene mutation causes the hemoglobin molecules in red blood cells to form a rigid chain. This causes the cells to stiffen into a sickle shape, clogging blood vessels and ...
It is a CRISPR-based medicine for sickle cell disease that partially silences BCL11A to boost the production of fetal hemoglobin, which is made with γ-globin. Blood cells typically stop making ...