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Hematopoietic stem cell-targeted gene therapy for sickle cell disease

Joint Research Seminar

Event Information

Date and Time 4/28/2021 (Wed) 9:30 ~ 10:15
Venue Online by Zoom (If you'd like to participate in this seminar, please contact aya-oka[at]ims.u-tokyo.ac.jp)
Speaker John F. Tisdale
Affiliation/Position Cellular and Molecular Therapeutics Branch (CMTB), National Heart, Lung, and Blood Institute (NHLBI), National Institutes of Health (NIH), Chief
Country United States of America
Title Hematopoietic stem cell-targeted gene therapy for sickle cell disease
Language English
Organizer Naoya Uchida

Overview

Sickle cell disease (SCD) is caused by a point mutation in the b-globin gene, resulting in anemia, pain, and organ damage. SCD can be cured by allogeneic hematopoietic stem cell (HSC) transplantation; however, a suitable donor is found for only ~10% of patients. Therefore, we developed HSC-targeted gene therapy with lentiviral gene transfer in SCD. In our clinical trial, a therapeutic b-globin gene is added to patient HSCs, allowing for one time cure of SCD. After accruing 47 patients, acute myeloid leukemia with chromosomal abnormalities and genetic mutations developed in a patient following gene therapy, unlikely due to lentiviral insertional mutagenesis. In this seminar, HSC-targeted gene therapy for SCD will be presented.