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St. Louis, Missouri – US Researchers USA They have used the CRISPR-Cas9 genome editor to correct the genetic defect of tungsten syndrome, a rare inherited disorder that leads to insulin deficiency in human stem cells. The stem cells differentiated into beta cells and were transplanted into mice whose beta cells had previously been destroyed by streptozocin. Animals were in the report. Medicine Translational Medicine (2020; 12: eaax9106) capable of producing insulin and regulating blood sugar.
Wolfram syndrome is a rare neurodegenerative disease with type I diabetes mellitus, diabetes insipidus, optic atrophy, and neurological symptoms. Around 300 cases have been described worldwide. The cause of the disease is a unique genetic defect in the WFS1 gene.
This makes patients candidates for treatment with the CRISPR-Cas9 gene editor. Since beta cells have already been destroyed, treatment must be carried out on stem cells, which will then differentiate and transplant.
A team led by Jeffrey Millman of the University of Washington School of Medicine in St. Louis / Missouri had managed to convert skin fibroblasts into pluripotent stem cells and then differentiate them into beta cells.
The researchers have now corrected the genetic defect in the stem cells of a patient with Wolfram syndrome and then differentiated the cells into beta cells. The beta cells were injected into mice whose beta cells had been destroyed by the streptozocin venom. The genetically engineered beta cells were able to produce insulin and cure diabetes in the animals.
A second group of mice received a beta cell transplant in which the genetic defect was not corrected. These animals were only able to produce insulin to a limited degree and soon became diabetic again.
Patients with Wolfram syndrome were not treated. It remains to be seen if the treatment would benefit the patient. Treatment can only correct diabetes. The other consequences of the disease were maintained.
Millman hopes the treatment will also help other patients with genetic diabetes mellitus. However, for most patients, insulin deficiency is the result of an autoimmune disease. A beta cell transplant generated from stem cells could probably only temporarily correct diabetes in these patients, since the transplanted cells would soon be recognized again by autoantibodies and destroyed by immune cells. © © wil / aerzteblatt.de
