CRISPR-aided gene therapy can treat many diseases like diabetes, experts say

CRISPR and diabetes. Can the latest world-beating technology beat a disease that afflicts so many?

Scientists are only beginning to unlock the potential of the CRISPR gene editing tool, which allows for fast and accurate modification of an organism’s genetic code. And while the ethical debates surrounding its use continue, the technique is finding more applications in fields like medicine and crop science.

Now, a study from researchers at the University of Chicago Medical Center shows that CRISPR-aided skin transplants can provide safe and minimally invasive options for the treatment of a range of human diseases. The scientists demonstrated their technique by grafting genetically altered skin onto mice, which prevented them from developing diabetes and obesity.

Here’s how it works. The research team started with the gene for a hormone called glucagon-like peptide 1 (GLP1) which stimulates the pancreas to produce insulin, which regulates blood sugar levels, and also decreases appetite by delaying gastric emptying. Using CRISPR, the team inserted a mutation into GLP1 that extends the hormone’s half-life in the blood stream and fixed a mechanism to increase production of insulin whenever cells are exposed to the antibiotic doxycycline.

The altered genes were inserted into skin cells which were then cultured in the lab to create enough material for a skin graft, which was performed on mice with intact immune systems. The researchers fed a high fat diet to both the genetically-altered mice and a control group, with the results showing that the GLP1-aided mice had less weight gain along with lowered glucose levels and reduced insulin resistance.

The research shows the clinical potential of “long-lasting, safe and versatile gene therapy approaches” based on skin stem cell transplantation, say the study’s authors, who state that there are many advantages to skin cell therapy as an approach to genetic engineering. The skin is the largest, most accessible organ of the body, skin cells grow quickly in lab cultures and skin grafts are both easily monitored and quickly removed if necessary. Further, skin stem cell transplants have a long and proven track record, with multiple decades of the safe and successful use of lab-grown tissue to treat burn victims, for example.

“We think this can provide a long-term safe option for the treatment of many diseases,” says Xiaoyang Wu, assistant professor with the Ben May Department for Cancer Research at the University of Chicago, in a press release. “It could be used to deliver therapeutic proteins, replacing missing proteins for people with a genetic defect, such as hemophilia. Or it could function as a metabolic sink, removing various toxins.”

Aiding in the treatment of diabetes and obesity alone would be major breakthroughs, though, as both are major health concerns worldwide. About 3.4 million Canadians are diabetic, with that number expected to grow to five million by the year 2025, representing 12.1 per cent of the population, according to Diabetes Canada. A further 5.7 million Canadians over the age of 20 are estimated to have prediabetes. One of the primary risk factors for both type 2 diabetes and prediabetes is being overweight.

The new research is published in the journal Cell – Stem Cell.