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A groundbreaking medical advancement has been achieved as a 42-year-old man with type 1 diabetes became the first person to produce insulin independently after receiving a transplant of genetically edited pancreatic islet cells. This innovative procedure was conducted without the use of immunosuppressant drugs, marking a significant step forward in the treatment of type 1 diabetes and potentially other autoimmune diseases.
The patient, who has been living with diabetes since the age of five, received the islet cell transplant through injections into the muscle of his forearm. Over a period of 12 weeks, the transplanted cells demonstrated their ability to secrete insulin in response to increased glucose levels following meals. Notably, the patient’s immune system did not attack the newly introduced cells, a common challenge in islet cell transplantation.
This new therapy builds upon existing methods that involve transplanting healthy islet cells into individuals whose own insulin-producing cells have been destroyed by their immune response. Traditionally, such transplants require the use of powerful immunosuppressant drugs to prevent rejection, which can lead to severe side effects, including increased infection risk.
In this pioneering study, researchers utilized CRISPR gene-editing technology to modify three specific aspects of the donor cells. Two of these edits reduced the expression of antigens that typically trigger an immune response, while a third enhancement increased the production of a protein known as CD47, which serves as a protective “do-not-eat” signal to the immune system.
While not all edited cells survived, the study found that those with successful triple edits remained functional and continued to produce insulin, confirming the potential effectiveness of this approach.
This research follows earlier advancements in diabetes treatment, including a case in 2023 where a woman with type 1 diabetes was able to produce insulin using stem cell-derived cells. However, that study did not address the immune rejection issue that this new research seeks to resolve.
The findings from this early-stage human trial provide promising evidence that CRISPR-edited islet cells can evade immune attacks while restoring insulin production. Researchers believe that this innovative approach could extend beyond diabetes, paving the way for safer cell therapies for a variety of chronic diseases without the complications associated with immunosuppressant medications. The study is published in The New England Journal of Medicine.
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