Diabetes reverted in mice with CRISPR—Cas9 gene-edited cells from patients
iPSCs from a patient with Wolfram syndrome were programmed into insulin-producing cells, which alleviated diabetes when transplanted into mice.
iPSCs from a patient with Wolfram syndrome were programmed into insulin-producing cells, which alleviated diabetes when transplanted into mice.
A research team from Washington University School of Medicine (MO, USA) transformed skin cell-derived induced pluripotent stem cells (iPSCs), from a patient with Wolfram syndrome, into insulin-producing cells. CRISPR—Cas9 gene editing was used to correct the mutation causing Wolfram syndrome in the human stem cells. The edited cells were then implanted into laboratory mice and found to reverse diabetes. The findings, published inScience Translational Medicine,may hold promise as a treatment for diabetes.
In this study, the research team derived beta cells from patients with Wolfram syndrome and used CRISPR—Cas9 gene-editing to edit a mutation in WFS1, the gene that causes Wolfram syndrome. The gene-edited cells were then compared to insulin-secreting beta cells from the same batch of iPSCs that had not been edited with CRISPR.
It was found that beta cells that were edited with CRISPR more efficiently secreted insulin in response to glucose, both in the test tube and in mice with a severe form of diabetes. Signs of diabetes disappeared in the mice that received the CRISPR-edited cells, implanted beneath the skin, with the blood sugar levels remaining within a normal range for the six-month period they were monitored. In contrast, mice that received unedited beta cells remained diabetic.
Ref:https://www.regmednet.com/diabetes-reverted-in-mice-with-crisprcas9-gene-edited-cells-from-patients/
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