Type 1 Diabetic Produces His Own Insulin After Gene-Edited Cell Transplant

Type 1 Diabetic Produces His Own Insulin After Gene-Edited Cell Transplant

A patient with type 1 diabetes has become the first in the world to produce his own insulin via transplanted cells edited with CRISPR.

The edits halted his own immune system from attacking the cells, leading to production of insulin in the pancreas as if he never had the disease in the first place.

Unlike the rampant type 2 diabetes, type 1 manifests as an autoimmune disorder whereby the patient’s immune system attacks and destroys a type of cell created in the pancreas to produce insulin, known as islet cells. 9.5 million people worldwide suffer from type 1 diabetes and there is no cure.

The standard of care beyond daily insulin injections involves using donated islet cells to allow a patient’s metabolism to function normally, followed by medications used to prevent the immune system from attacking those cells. These medications have harmful side effects.

A team of biomedical researchers primarily from Uppsala University, Sweden, in collaboration with UC San Francisco, successfully used the CRISPR gene-editing method to take a donor’s islet cells and modify them so as to evade prosecution by the patient’s immune system.

Three changes in total to the genetic code were needed to ensure the transplanted donor cells didn’t trigger an immune response, including changes to cell membrane signal proteins used as a sign of attack by white blood cells.

CRISPR TO THE RESCUE:

• Infant With Incurable Disease is First to Successfully Receive Personalized Gene Therapy Treatment
• Aggressive Leukemia Disappears in 13-Year-old Girl Who was First to Receive New CRISPR Treatment
• First Patient to Receive Gene Therapy ‘Cure’ for Beta-Thalassemia Living Pain-Free

As a preliminary study, the man received only a small amount of the donated, modified cells, meaning he still requires daily insulin injections. However the results show that the cells produced their own insulin and remained unperturbed by the immune system.

After 12 weeks, the cells were still producing endogenous insulin, setting the stage for longer-term and more robust experiments in the future.

The study was published in the New England Journal of Medicine.