What Gene Editing Could Mean for Type 1 Diabetes


Altering human genetics is a sensitive subject. There are a lot of things that could potentially go wrong, but also many that could go right. CRISPR/Cas9 technology allows scientists to precisely cut out a segment of DNA and replace it with a new segment. By modifying specific genes, they could essentially eliminate certain diseases and remove inherited diseases from the human germline.

This unleashes new opportunities when it comes to treating – and potentially curing – diabetes. Scientists recently implanted skin grafts with a gene (GLP1) to stimulate insulin secretion by the pancreas. They attached these grafts to mice and found that the new genes helped to remove excess glucose from the bloodstream. Using skin grafts is a safe and relatively inexpensive process.

Researchers in Sweden managed to use CRISPR/Cas9 to switch off an enzyme that is involved in regulating the TXNIP gene which affects beta cell death and decreases insulin production. In Australia, the technology was used to try to identify rogue immune cells that attack the pancreas and contribute to the development of type 1 diabetes.

However, there is still more research that needs to be done to fully understand the impact of gene editing and potential effects that it could have. Though highly precise, there is still around a one percent chance of off-target effects occurring. These are changes to other parts of the genome outside of the area targeted by CRISPR/Cas9. There is a lot of risks involved with changing human DNA and many questions that are still unanswered. Furthermore, many of these studies have been conducted on mice and results do not always correlate exactly to humans.

But with more extensive testing and research, scientists may be able to find a safe way to treat or even cure diabetes through gene editing. Studies that exist so far hold potentially promising results. It is these types of cutting-edge, innovative approaches that could change the future of type 1 diabetes. The Diabetes Research Connection proudly supports early career scientists in pursuing novel research for type 1 diabetes. Click to learn more about current projects and provide support.

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