Speaker: Professor WU Yuxuan, School of Life Sciences, East China Normal University (ECNU)
Date and time: 13:30-15:00, Nov.11 (Friday)
Venue: Auditorium at L Building
Abstract:
Gene editing to disrupt the GATA1-binding site at the +58 BCL11A erythroid enhancer could induce γ-globin expression, which is a promising therapeutic strategy to alleviate ß-hemoglobinopathy caused by HBB gene mutation. In the present study, we report the preliminary results of an ongoing phase 1/2 trial (NCT04211480) evaluating safety and efficacy of gene editing therapy in children with blood transfusion-dependent ß-thalassemia (TDT).
