Diabetes Reversed in Mice for the First Time

Apr 22, 2020 by Iris Dawn Tabangcora


This year dawns with a massive shot at discovering a potential cure for diabetes as scientists from Washington University School of Medicine in St. Louis were able to cure the disease in mice for the first time.

Previously thought of as incurable, millions of people worldwide are left to deal with strict blood sugar monitoring and multiple drug therapies on a daily basis. This may no longer be the case in the years to come because of advances in genetic editing.



Diabetes refers to a group of metabolic disorders that share the same characteristic of high sugar levels in the blood. This is caused by a complex interaction of genetics and environmental factors. The body struggles to optimize glucose utilization, control glucose production, and enhance insulin secretion. If left untreated, this will cause multi-organ failure and early death. In fact, it is the leading cause of adult blindness, non-traumatic lower extremity amputations, and chronic kidney disease.

It continues to be a pressing public health issue and is a significant cause of morbidity and mortality. Thus, decades of intensive research have been dedicated to understanding its pathology on a molecular basis. This has given birth to a host of new and improved therapies while a definitive cure is still not available.

In this study, researchers used induced pluripotent stem cells from the skin of a patient with Wolfram Syndrome, a rare form of insulin-requiring diabetes caused by a single gene mutation. Conceptually, this kind of mutation is easier to fix. These stem cells were converted into insulin-producing cells and underwent genetic editing to correct the genetic defect responsible for the syndrome using CRISPR-Cas9 technology.

These insulin-producing cells were implanted into mice with pre-existing diabetes and blood sugar levels high enough to be fatal in humans. The result? Blood sugar levels normalized in as early as two weeks and were maintained for as long as 9-12 months. This medical breakthrough is a dramatic stepping stone towards curing more common forms of diabetes like type 1 and 2.

Earlier, researchers were also able to discover how to convert stem cells into insulin-producing cells but the process was not as effective as some of the cells turned out to be liver, neuronal, and heart cells. While not exactly harmful, these off-target cells meant less therapeutically relevant cells. On the other hand, the cells that underwent CRISPR-Cas9 technology were able to control blood sugar more effectively. In fact, the corrected insulin-producing cells were indistinguishable from the ones made from stem cells of healthy people with no diabetes.

The researchers claimed that eventually, the process of converting insulin-producing cells from stem cells would be easier and less intrusive. They are also looking at developing stem cells from urine samples. This will pave the way for personalized regenerative gene therapy.

It's still a long way for this prospective cure to be declared as safe in humans. It will need more testing on larger animals and for longer periods of time. Still, this proves to be a massive breakthrough in the search for a cure.

This study is published online and can be accessed in the journal Science Translational Medicine.

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