Gene Edit Turns Pancreatic Cells Into Insulin Factories

Researchers at Harvard Medical School have genetically reprogrammed pancreatic ductal cells to produce and release insulin in response to high blood sugar. The work, published in Science Translational Medicine, could offer a new route to treating diabetes, which affects an estimated 830 million people worldwide.

A healthy pancreas contains about a billion beta cells, the primary producers of insulin. In people with diabetes, these cells are either missing or dysfunctional. Ductal cells, which normally line the pancreas, occasionally transform into beta cells on their own—an unusual event for adult cells. The team used a genetic screen to find genes involved in that transformation.

Removing the gene ALDH3B2 increased the rate at which ductal cells became beta-like cells from under 1 percent to about 8.5 percent. The experiments were first done on human cells in a dish and then transplanted into mice with diabetes. Human insulin circulated in the mice, and their glucose levels dropped to near-normal levels for six weeks.

The exact mechanism by which ALDH3B2 affects the conversion is not yet known. Because the gene is used by many cells in the body, precision will be critical to avoid complications. Next steps could involve gene therapy or finding small molecules to inhibit the gene.

Other treatments aim to generate new insulin-producing cells in the lab and transplant them. A clinical trial launched earlier this year is testing a gene therapy that gives muscle cells instructions to make insulin. The story originally appeared on WIRED en Español and was translated from Spanish, as reported by wired.com.