Base-Edited CAR7 T Cells for Relapsed T-Cell Acute Lymphoblastic Leukemia
Robert Chiesa1, Christos Georgiadis1, Farhatullah Syed1
1From Great Ormond Street Hospital for Children NHS Trust (R.C., G.O., T.B., J.C., S.A., R.T., K.G., D.O., A.V., W.Q.) and the UCL Great Ormond Street Institute of Child Health (C.G., F.S., H.Z., A.E., S.A.G., R.P., A.K., W.Q.) - both in London.
The New England Journal of Medicine
|June 14, 2023
Summary
Base editing precisely converts DNA nucleotides, enabling gene inactivation without breaks. This CRISPR-based approach generated effective chimeric antigen receptor T cells for treating relapsed leukemia in children.
Area of Science:
- CRISPR-mediated cytidine deamination for precise DNA base editing.
- Development of universal, off-the-shelf chimeric antigen receptor (CAR) T cells.
Background:
- Cytidine deamination via CRISPR enables targeted C-to-T conversion without DNA breaks.
- This precise gene editing avoids chromosomal aberrations, making it suitable for therapeutic applications.
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