...in Brief Hematology Thalassemia α globin (HBA) Cell culture and patient sample studies suggest inhibiting HBA-MCS-R2...
...HBB) to help treat β-thalassemia. University of Oxford Preclinical News α globin MCS-R2 enhancer sequence (HBA-MCS-R2...

...INDICATION: Thalassemia Cell culture and patient sample studies suggest inhibiting HBA-MCS-R2 could help treat β-thalassemia. In...
...could include testing HBA-MCS-R2 inhibition in animal models of β-thalassemia. TARGET/MARKER/PATHWAY: α-globin MCS-R2 enhancer sequence (HBA-MCS-R2...
...doi:10.1038/s41467-017-00479-7 CONTACT: Douglas R. Higgs, University of Oxford, Oxford, U.K. email: doug.higgs@imm.ox.ac.uk Elizabeth S. Eaton Alpha globin (HBA) Alpha globin MCS-R2 enhancer sequence (HBA-MCS-R2) Beta...

...Communications , researchers at the University of Oxford and colleagues showed that deletion of the alpha globin MCS-R2 enhancer sequence (HBA-MCS-R2)...
...CD34+ human erythroid cells, the researchers showed that CRISPR- Cas9 (CRISPR-associated protein 9) deletion of HBA-MCS-R2...
...of the HBB gene. In CD34+ cells from patients with beta thalassemia, CRISPR-Cas9 deletion of HBA-MCS-R2...