A new study published in the journal Science Translational Medicine finds that diabetes develops during childhood or adolescence in patients with Wolfram syndrome and rapidly require insulin-replacement therapy, in which insulin injections are injected multiple times every day. Some other issues with vision or balance may also develop in them. Wolfram syndrome also causes early death.
Researchers used stem cells that are produced from the skin of patients with an uncommon, genetic form of insulin-dependent diabetes called Wolfram syndrome and introduce them into the cells that produce insulin and then the gene-editing tool CRISPR-Cas9 was used to correct the syndrome causing a genetic defect. After this, the cells were implanted into the lab mice and the unrelenting diabetes was relieved from those mice.
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The researchers at Washington University School of Medicine in St. Louis recommends that the CRISPR-Cas9 technique may hold guarantee as a treatment for diabetes, especially the forms brought about by a single gene mutation and it might be helpful one day in certain patients with the more common types of diabetes like type 1 and type 2.
An assistant professor of medicine and biomedical engineering at Washington University, a co-senior investigator Jeffrey R. Millman tells that this happened the first time that CRISPR technique is used to fix the diabetes-causing genetic defect and successfully reverse diabetes. In this study, the researchers utilized cells from a patient with Wolfram syndrome because it would be simpler to correct a genetic defect caused by a single gene. But they consider it a stepping stone toward applying gene therapy to several patients with diabetes.
In this examination, the researchers made the extra steps of getting these cells from patients and used the CRISPR-Cas9 technique on those cells to correct the gene mutations that cause Wolfram syndrome (WFS1). After this, the analysts compared the gene-edited cells to insulin-secreting beta cells from the stem cells on which the CRISPR technique wasn’t used.
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In the test tube and mice with an extreme type of diabetes, the recently developed beta cells that were edited with CRISPR more effectively secreted insulin because of glucose. Diabetes vanished rapidly in mice with the CRISPR-edited cells implanted underneath the skin, and the glucose levels in animals remained in the ordinary range for a half year they were observed. Animals that were receiving unedited beta cells remained diabetic. Insulin could be produced from their recently implanted cells, just not enough to reverse their diabetes.
Researchers hope that in future CRISPR might help to correct mutations in beta cells in patients whose diabetes is the result of numerous environmental and heredity factors like type 1 and type 2 diabetes.
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Millman tells that researchers are excited because they had the option to combine both technologies, CRISPR that was helpful to correct genetic defects and growing beta cells from induced pluripotent stem cells. They found that the beta cells produced from the stem cells of healthy individuals with no diabetics were indistinguishable from corrected beta cells.
Urano stated that in the future it might be possible to take urine from the patients to make stem cells that can develop into beta cells, using CRISPR correct mutations in the cells, transplant again into the patients, and reverse diabetes in their clinics. Genetic testing in diabetes patients will help to distinguish genes that will lead to personalized regenerative gene therapy.
