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, according to results published in the journal Science Translational Medicine.
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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