20:53 , Oct 4, 2017 |  BC Innovations  |  Translation in Brief

New Therapeutic Targets and Biomarkers: September 2017

New Therapeutic Targets and Biomarkers: September 2017 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during September 2017. Therapeutic targets are defined as any protein, gene or other molecule...
18:17 , Sep 27, 2017 |  BC Innovations  |  Distillery Therapeutics

Hematology

INDICATION: Thalassemia Cell culture and patient sample studies suggest inhibiting HBA-MCS-R2 could help treat β-thalassemia. In patients, high levels of α-globin (HBA) are associated with disease severity. In human erythroid progenitor cells, CRISPR-Cas9 (CRISPR-associated protein...
21:19 , Sep 8, 2017 |  BC Extra  |  Preclinical News

Deletion of alpha globin enhancer could treat beta thalassemia

In a paper published in Nature Communications, researchers at the University of Oxford and colleagues showed that deletion of the alpha globin MCS-R2 enhancer sequence (HBA-MCS-R2) reduced alpha globin (HBA) expression and corrected the imbalance...