A new treatment has emerged for 'Type 1 diabetes', a congenital genetic disease that affects 9 million people worldwide.
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korocamia@naver.com | 2025-09-07 16:40:42
It is expected to significantly improve the quality of life for patients as they may no longer need to take diabetes medication for the rest of their lives.
The international academic journal 'Nature' reported on the 6th (local time) that Sana Biotechnology, a biotech company in Washington State, USA, used the gene-editing technology 'CRISPR' to create 'pancreatic islet beta cells' that secrete insulin without any issues and transplanted them into a patient with Type 1 diabetes. A 42-year-old man, who had been suffering from Type 1 diabetes for 37 years since its onset at age 5, received the beta cell transplant and has been secreting insulin normally for over three months without needing diabetes medication.
Beta cells in the pancreatic islets are responsible for secreting insulin, a hormone that regulates blood sugar, and controlling blood sugar levels. Type 1 diabetes is an autoimmune disease where immune cells mistakenly identify the insulin-producing pancreatic islet beta cells as 'enemies' and attack them. Unlike Type 2 diabetes, which is acquired due to diet or lifestyle habits, it is a congenital genetic disease that mainly develops in children and young adults under the age of 20. Once diagnosed, like Type 2 diabetes, patients must continuously take diabetes medication.
Sana Biotechnology's idea was to edit the genes of the pancreatic islet beta cells so that they would not be recognized as 'enemies' by the immune cells. All cells have a function to signal to immune cells that they are not enemies. They display a protein that acts as a 'name tag' to prevent immune cells from attacking them. Autoimmune diseases primarily occur when there is an error in this process.
Sana Biotechnology used CRISPR to remove two genes responsible for this function in the pancreatic islet beta cells and introduced a protein (CD47) gene that sends a 'do not attack me' signal. Through genetic manipulation, they created a pathway for the cells to evade the attack of immune cells.
The industry expects that this new treatment will provide a new therapeutic option for patients with Type 1 diabetes. The newly developed treatment allows for normal insulin secretion without the need for diabetes medication or immunosuppressants.
Tim Kieffer, a professor at the University of British Columbia in Vancouver, Canada, expressed that more data is needed to confirm its efficacy but evaluated it as "an important milestone that could enable an effective treatment without immunosuppression."
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