PubMed · 2026-04-23
Researchers review how CRISPR gene editing and gene therapy could cure Type 1 Diabetes by regrowing insulin-producing cells and retraining the immune system to stop attacking them, summarizing five ongoing clinical trials and the key hurdles still to overcome.
Five active clinical trials (including NCT03162237 and NCT06938334) are testing CRISPR-edited cell replacement and immune-modulation strategies, with early-phase data confirming feasibility and acceptable safety profiles.
Two main genetic targets — PD-L1 (immune checkpoint) and FOXP3 (regulatory T-cell master gene) — are being used to suppress the autoimmune attack without broadly disabling the immune system.
Critical unresolved risks include CRISPR off-target edits, insertional mutagenesis from viral gene delivery, and infection/rejection concerns tied to pig-to-human (xenotransplantation) cell sources.