STM: Genetic modification of stem cells can improve diabetes

According to a recent study, researchers used cells extracted from the skin of a patient with rare insulin-dependent diabetes mellitus (Wolfram syndrome) to induce pluripotent stem cells and convert them into insulin-secreting cells. The editing tool CRISPR-Cas9 corrects the genetic defects that cause the syndrome. They then implanted these cells in mice and cured the mice 0010010 #39;s diabetes.

This research from researchers at the University of Washington School of Medicine in St. Louis suggests that the CRISPR-Cas9 technology may serve as a powerful weapon for the treatment of diabetes, especially diabetes caused by a single gene mutation. The study was published online in the journal Science Translational Medicine on April 22.

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Patients with Wolfram syndrome develop diabetes during childhood or adolescence, and quickly require insulin replacement therapy, requiring multiple injections of insulin daily. Most patients experience vision and balance problems and other problems. In many patients, the syndrome can lead to early death.

0010010 quot;This is the first time that CRISPR has been used to repair diabetes caused by a genetic defect, 0010010 quot; said co-investigator Dr. Jeffrey R. Millman, an assistant professor of medicine and biomedical engineering at the University of Washington.

Wolfram syndrome is caused by mutations in a single gene, so the researchers hope to understand that by combining stem cell technology with CRISPR, diabetes caused by mutations can be corrected.

A few years ago, Millman and colleagues discovered how to transform human stem cells into pancreatic beta cells. The researchers have developed a new technology that can more effectively transform human stem cells into beta cells, which have a fairly good effect in controlling blood sugar.

In this study, they took additional steps to extract these cells from patients and use CRISPR-Cas9 gene editing tools on these cells to correct the genetic mutations that caused Wolfram syndrome (WFS1). The researchers then compared these gene-edited cells with insulin-secreting beta cells from the same batch of stem cells that had not been edited with CRISPR.

The author implanted CRISPR-edited cells into mice with diabetes by subcutaneous implantation. The results showed that diabetes quickly disappeared, and the animal ’s blood sugar levels remained normal throughout the six months of monitoring. Within range. In contrast, mice that received unedited beta cells still had diabetes.

In the future, using CRISPR to correct certain mutations in beta cells may help those with diabetes caused by multiple genetic and environmental factors. 0010010 quot;We are excited that we are able to combine these two technologies and use CRISPR to correct genetic defects, 0010010 quot; Millman said. 0010010 quot;In fact, we found that the corrected beta cells are no different from those derived from the stem cells of healthy people without diabetes. 0010010 quot;

The researchers said that in the future, the process of making β cells from stem cells should become easier. For example, scientists have developed less invasive methods to make induced pluripotent stem cells from the blood-they are working on developing stem cells from urine samples. (from Bioon.com)