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.