Cell therapy of the GLUT1-DS using directed GLUT1 engineered endothelial cell progenitors

The GLUT1-DS is cause by genetic mutation of the gene encoding for Glut1. In the era of gene therapy this defect can be corrected but genetic manipulation and the use of viral vectors brings risks. A more conservative and safer approach is to perform genetic correction in lab on cells isolated from the patients. Once corrected cells are given back to patients with an injection. Our approach named cell therapy allow to isolate precursors of the blood brain barrier from a blood sample, correct the genetic defect only in these cells in laboratory and use the corrected cells to restore the glucose flux across the blood brain barrier. Using this approach, no viral vectors will be injected into patients. We believe this approach brings promise to solve the clinical problem while minimizing risks and unpredictable side effects of a gene therapy.