Diabetic man with gene-edited cells now produces insulin without needing transplant drugs

Man with type 1 diabetes produces insulin from gene-edited cells, avoiding transplant drugs.

: A groundbreaking study demonstrates the use of CRISPR to modify pancreatic cells for insulin production in type 1 diabetes patients without immunosuppressive drugs. Conducted by scientists in Sweden and the U.S., the research focused on a 42-year-old man who received the gene-edited islet cells. These cells were altered to reduce immune response, containing three specific edits, and showed promising results in insulin production. However, this is a preliminary success and more trials are needed to assess long-term viability and effectiveness.

In a groundbreaking proof-of-concept study published in The New England Journal of Medicine on August 8, 2025, a 42-year-old man with long-standing type 1 diabetes received a transplant of insulin-producing pancreatic islet cells genetically edited with CRISPR. These “hypoimmune” cells were engineered to delete HLA class I and II antigens and overexpress CD47, enabling them to evade immune detection and removing the need for lifelong immunosuppressive drugs typically required after such transplants.

The patient underwent seventeen separate injections of the modified cells into his forearm beginning in December 2024. At 12 weeks, MRI scans confirmed the survival of the transplanted cells, and blood tests detected C-peptide, indicating that the cells were actively producing insulin. At six months, the cells continued to function and responded to a mixed-meal tolerance test without triggering immune rejection.

Despite these promising results, the patient still needs external insulin because the transplanted dose amounted to only about 7 percent of what would be necessary for full insulin independence. The trial was intended to assess safety and feasibility rather than deliver a complete cure.

Experts have described the findings as a significant early step toward functional cures for type 1 diabetes, though they stress the importance of further studies to verify long-term safety, effectiveness, scalability, and affordability. They also highlight the potential for future stem cell-derived, gene-edited therapies to overcome current donor shortages.

This is the first documented case in humans where gene-edited donor islets have produced insulin without the use of immunosuppression, marking a milestone in regenerative medicine and pushing the field closer to developing viable, long-term treatments for type 1 diabetes.

Sources: The New England Journal of Medicine, Medscape Medical News, Gizmodo, Uppsala University

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