Patent Portfolio
Model of congenital urea cycle disorders and utilization thereof
| Ref. No. | AJ246 |
|---|---|
| Appl. No. （filing date） |
PCT/JP2021/011317（2021/3/19） |
| Pub. No. （Pub. date） |
WO/2021/193408（2021/9/30） |
| Priority claim No. （Priority date） |
JP2020-051532（2020/03/23） |
| Inventor | OSAFUNE, Kenji, et al |
| Assignee | Kyoto University |
| Title of Invention | Model of congenital urea cycle disorders and utilization thereof |
| Abstract | This invention provides a method for screening or evaluating a therapeutic drug for congenital urea cycle disorders, comprising a step for contacting a drug candidate substance with hepatocytes, which have been obtained by inducing the differentiation of iPS cells obtained from somatic cells derived from a patient of congenital urea cycle disorders, and measuring the urea cycle activity of and/or fat accumulation in the hepatocytes. |
| Information (External link) |
https://www.j-platpat.inpit.go.jp/c1800/PU/WO-A-2021-193408/C3E52D35ADCA3E5725EAA60E9CCE829CDE602360FC989540544F31CCE0B94E98/50/en |