27 Apr CRISPR-Edited Stem Cells Cured Diabetes in a Mouse Model
MedicalResearch.com Interview with:
Fumihiko Urano, MD, PhD
Samuel E. Schechter Professor of Medicine
Division of Endocrinology, Metabolism, and Lipid Research
Washington University School of Medicine
MedicalResearch.com: What is the background for this study?
Response: Wolfram syndrome is a rare monogenic disease characterized by insulin-dependent diabetes, retinal degeneration, and neurodegeneration. Using gene editing by CRISPR-Cas9, in combination with patient-derived induced pluripotent stem cells (iPSCs), we were able to make normal insulin-producing pancreatic beta cells by correcting Wolfram Syndrome gene mutation.
We could cure diabetes in cells and mice. Because we can create any types of tissues from iPSCs, our next step would be to replicate this success for other medical problems, including retinal regeneration and neurodegeneration.
MedicalResearch.com: What should readers take away from your report?
Response: It’s a proof of concept demonstrating that correcting gene defects that cause or contribute to diabetes — in this case, in the Wolfram syndrome gene — we can make beta cells that more effectively control blood sugar. It’s also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration.
MedicalResearch.com: What recommendations do you have for future research as a result of this work?
Response: Our study suggests that we may be able to cure monogenic diseases, especially monogenic diabetes (i.e., diabetes caused by gene mutations) and monogenic retinal degeneration, using gene editing in combination with patient-derived iPSCs. Genetic testing in patients with diabetes and retinal degeneration will guide us to identify genes that should be corrected, which will lead to a personalized regenerative gene therapy.
MedicalResearch.com: Is there anything else you would like to add?
Response: My deepest thanks and gratitude to my patients who donated their cells and my donors who have been supporting my research. I hope I can treat my patients with diabetes using a personalized gene therapy in my clinic in the near future.
Kristina G. Maxwell, Punn Augsornworawat, Leonardo Velazco-Cruz, Michelle H. Kim, Rie Asada, Nathaniel J. Hogrebe, Shuntaro Morikawa, Fumihiko Urano, Jeffrey R. Millman. Gene-edited human stem cell–derived β cells from a patient with monogenic diabetes reverse preexisting diabetes in mice. Science Translational Medicine, 2020; 12 (540): eaax9106 DOI: 10.1126/scitranslmed.aax9106
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