The current approach called ex vivo gene editing, involves:
- Isolating a patient's specialized cells called hematopoietic stem/progenitor cells.
- Editing these cells in a laboratory to correct the gene.
- Selecting the corrected cells.
- Multiplying the number of corrected cells.
- Transplanting them back into the patient.
Despite initial success, this approach is hindered by difficulties in processing stem cells, risks associated with removing these cells from the bone marrow, low efficiency of cell survival after the corrected cells are returned to the body, and the high cost of individualized therapy.
The team will develop a gene editing technology that corrects stem cells in the bone marrow without removing the cells outside the body.
They will combine nanotechnology and synthetic biology in a way that the correction can be specifically applied to the targeted stem cells.
Their goal is to provide an effective and low-cost cure for hematological diseases like sickle cell disease and β-thalassemia.
The R21 award will provide $420,750 over two years. For this project, Tong and his collaborators received preliminary funding from the University of Kentucky Ignite Research Collaborations.
Research reported in this publication was supported by the National Heart Lung and Blood Institute of the National Institutes of Health under Award Number R21HL166178. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.