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Genotype-phenotype correlation analysis and therapeutic development using a patient stem cell-derived disease model of Wolfram syndrome
Rie Asada Kitamura, Kristina G. Maxwell, Wenjuan Ye, Kelly Kries, Cris M Brown, Punn Augsornworawat, Yoel Hirsch, Martin M Johansson, Tzvi Weiden, Joseph Ekstein, Joshua Cohen, Justin Klee, Kent Leslie, Anton Simeonov, Mark J. Henderson, Jeffrey R. Millman, View ORCID ProfileFumihiko Urano
doi: https://doi.org/10.1101/2021.11.07.467657
Rie Asada Kitamura
1Department of Medicine, Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA
Kristina G. Maxwell
1Department of Medicine, Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA
3Department of Biomedical Engineering, Washington University in St. Louis, 1 Brookings Drive, St. Louis, MO 63130, USA
Wenjuan Ye
4National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, Maryland, 20850, USA
Kelly Kries
1Department of Medicine, Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA
Cris M Brown
1Department of Medicine, Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA
Punn Augsornworawat
1Department of Medicine, Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA
3Department of Biomedical Engineering, Washington University in St. Louis, 1 Brookings Drive, St. Louis, MO 63130, USA
Yoel Hirsch
5Dor Yeshorim, Committee for Prevention of Jewish Genetic Diseases, Brooklyn, NY 11211, USA
Martin M Johansson
5Dor Yeshorim, Committee for Prevention of Jewish Genetic Diseases, Brooklyn, NY 11211, USA
Tzvi Weiden
6Dor Yeshorim, Committee for Prevention of Jewish Genetic Diseases, Jerusalem, Israel
Joseph Ekstein
5Dor Yeshorim, Committee for Prevention of Jewish Genetic Diseases, Brooklyn, NY 11211, USA
Joshua Cohen
7Amylyx Pharmaceuticals, Cambridge, MA 02141, USA
Justin Klee
7Amylyx Pharmaceuticals, Cambridge, MA 02141, USA
Kent Leslie
7Amylyx Pharmaceuticals, Cambridge, MA 02141, USA
Anton Simeonov
4National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, Maryland, 20850, USA
Mark J. Henderson
4National Center for Advancing Translational Sciences, National Institutes of Health, Rockville, Maryland, 20850, USA
Jeffrey R. Millman
1Department of Medicine, Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA
3Department of Biomedical Engineering, Washington University in St. Louis, 1 Brookings Drive, St. Louis, MO 63130, USA
Fumihiko Urano
1Department of Medicine, Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA
2Department of Pathology and Immunology, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO 63110, USA
Posted November 08, 2021.
Genotype-phenotype correlation analysis and therapeutic development using a patient stem cell-derived disease model of Wolfram syndrome
Rie Asada Kitamura, Kristina G. Maxwell, Wenjuan Ye, Kelly Kries, Cris M Brown, Punn Augsornworawat, Yoel Hirsch, Martin M Johansson, Tzvi Weiden, Joseph Ekstein, Joshua Cohen, Justin Klee, Kent Leslie, Anton Simeonov, Mark J. Henderson, Jeffrey R. Millman, Fumihiko Urano
bioRxiv 2021.11.07.467657; doi: https://doi.org/10.1101/2021.11.07.467657
Genotype-phenotype correlation analysis and therapeutic development using a patient stem cell-derived disease model of Wolfram syndrome
Rie Asada Kitamura, Kristina G. Maxwell, Wenjuan Ye, Kelly Kries, Cris M Brown, Punn Augsornworawat, Yoel Hirsch, Martin M Johansson, Tzvi Weiden, Joseph Ekstein, Joshua Cohen, Justin Klee, Kent Leslie, Anton Simeonov, Mark J. Henderson, Jeffrey R. Millman, Fumihiko Urano
bioRxiv 2021.11.07.467657; doi: https://doi.org/10.1101/2021.11.07.467657
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