Article Synopsis
- The study explores the transplantation of genetically modified allogeneic donor islet cells in a man with type 1 diabetes without using immunosuppressive drugs, addressing the risk of immune rejection.
- The cells were edited using CRISPR technology and then implanted into the participant's forearm muscle, demonstrating no immune response after 12 weeks.
- Measurements indicated stable insulin secretion, with only minor adverse events occurring that were unrelated to the cell transplant procedure.
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Article Abstract
The need to suppress a patient's immune system after the transplantation of allogeneic cells is associated with wide-ranging side effects. We report the outcomes of transplantation of genetically modified allogeneic donor islet cells into a man with long-standing type 1 diabetes. We used clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 12b (Cas12b) editing and lentiviral transduction to genetically edit the cells to avoid rejection; the cells were then transplanted into the participant's forearm muscle. He did not receive any immunosuppressive drugs and, at 12 weeks after transplantation, showed no immune response against the gene-edited cells. C-peptide measurements showed stable and glucose-responsive insulin secretion. A total of four adverse events occurred, none of which were serious or related to the study drug. (Funded by the Leona M. and Harry B. Helmsley Charitable Trust; EudraCT number, 2023-507988-19-00; ClinicalTrials.gov number, NCT06239636.).
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