Publications by authors named "Stephanie C Fowler"
Article Synopsis
- The study explores using CRISPR/Cas9 technology to disrupt gene regulatory elements that suppress the expression of fetal hemoglobin (HbF), which could be a new treatment for sickle cell disease and β-thalassemia.
- Researchers successfully targeted the BCL11A protein, which blocks γ-globin gene expression, and induced significant levels of HbF in modified hematopoietic cells.
- The modified cells showed no harmful off-target effects and maintained healthy differentiation into various blood cell types, suggesting the approach could be a safe and effective treatment for hemoglobin-related disorders.
