简单说明
In type 1 diabetes the immune system destroys the cells in the pancreas that make insulin. You can now grow replacement insulin-producing cells from stem cells — but transplant them and the same immune system destroys them again, unless the patient takes immune-suppressing drugs for life. Gene editing is being used to make the replacement cells invisible to the immune system, so that they can survive without those drugs.
深入了解
Type 1 diabetes is autoimmune beta-cell destruction; type 2 is a metabolic disease of insulin resistance with polygenic contribution and is not an editing target. Stem-cell-derived islet transplantation is clinically effective but requires chronic immunosuppression. Hypoimmune engineering — knocking out HLA class I and II via B2M and CIITA, and overexpressing CD47 to evade natural killer cells — aims to allow allogeneic islet transplantation without immunosuppression. Sana Biotechnology reported early evidence of hypoimmune islet cell survival and insulin production without immunosuppression.
A different use of editing again
This site's disease pages fall into three groups: correct the gene, remove the harmful protein, or engineer the cells. Diabetes is squarely in the third. Nothing about the patient's genome is edited — the transplanted cells are. It is the same logic as allogeneic CAR-T, applied to a metabolic disease.
Sources
- New England Journal of Medicine · 2025
Survival of transplanted allogeneic beta cells with no immunosuppression ↗