Reversing diabetes in mice with CRISPR-edited stem cells

| | April 30, 2020
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Insulin injections can control diabetes, but patients still experience serious complications such as kidney disease and skin infections. Transplanting pancreatic tissues containing functional insulin-producing beta cells is of limited use, because donors are scarce and patients must take immunosuppressant drugs afterward.

Now, scientists at Washington University in St. Louis have developed a way to use gene editing system CRISPR-Cas9 to edit a mutation in human-induced pluripotent stem cells (iPSCs) and then turn them into beta cells. When transplanted into mice, the cells reversed preexisting diabetes in a lasting way.

While the researchers used cells from patients with Wolfram syndrome—a rare childhood diabetes caused by mutations in the WFS1 gene—they argue that the combination of a gene therapy with stem cells could potentially treat other forms of diabetes as well.

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“We can generate a virtually unlimited number of beta cells from patients with diabetes to test and discover new drugs to hopefully stop or even reverse this disease,” Jeffrey Millman, the study’s co-senior author, said in the video statement. “Perhaps most importantly, this technology now allows for the potential use of gene therapy in combination with the patient’s own cells to treat their own diabetes by transplantation of lab-grown beta cells.”

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