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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and Transfusion Dependent β-Thalassemia

Haydar Frangoul, MD, MS, Director of Stem Cell Transplant, Sarah Cannon Research Institute, The Children’s Hospital at TriStar Centennial

Website

A presentation of data on using gene addition and gene editing technology to treat patients with sickle cell disease and transfusion dependent beta thalassemia. I will also present preliminary data on our work using CRISPR-Cas9 to target BC11A and induced fetal hemoglobin production.

Speaker Bio

Haydar Frangoul, MD, MS, is the Director of the Pediatric Stem Cell Transplant program at Tristar Centennial Children’s Hospital and the Sarah Cannon Research Institute in Nashville Tennessee. Dr. Frangoul had leadership positions in the Children Oncology group, and Pediatric Blood and Marrow Transplant Consortium. His primary interest is allogeneic stem cell transplant using an alternative donor sources especially for non-malignant diseases. Dr Frangoul has authored or co-authored more than 130 peer reviewed manuscripts. He has been an invited speaker at numerous national and international conferences. He has been one of the leading investigators in a clinical trial using CRISPR-Cas9 gene editing for patients with sickle cell disease and transfusion dependent thalassemia.

GES Colloquium is jointly taught by Drs. Jen Baltzegar and Sumit Dhole, who you may contact with any class-specific questions. As conditions allow, colloquium will be held in-person in Poe 202, as well as live-streamed via Zoom (at least January will be 100% virtual). Please subscribe to the GES newsletter and Twitter for updates .