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- W4297241591 abstract "Abstract The urea cycle is a liver-based pathway enabling disposal of nitrogen waste. Urea cycle disorders (UCDs) are inherited metabolic diseases caused by deficiency of enzymes or transporters involved in the urea cycle and have a prevalence of 1:35,000 live births. Patients present recurrent acute hyperammonaemia, which causes high rate of death and neurological sequelae. Long-term therapy relies on a protein-restricted diet and ammonia scavenger drugs. Currently, liver transplantation is the only cure. Hence, high unmet needs require the identification of effective methods to model these diseases to generate innovative therapeutics. Advances in both induced pluripotent stem cells (iPSCs) and genome editing technologies have provided an invaluable opportunity to model patient-specific phenotypes in vitro by creating patients’ avatar models, to investigate the pathophysiology, uncover novel therapeutic targets and provide a platform for drug discovery. This review summarises the progress made thus far in generating 2- and 3-dimensional iPSCs models for UCDs, the challenges encountered and how iPSCs offer future avenues for innovation in developing the next-generation of therapies for UCDs." @default.
- W4297241591 created "2022-09-28" @default.
- W4297241591 creator A5020800689 @default.
- W4297241591 creator A5031953037 @default.
- W4297241591 date "2022-09-26" @default.
- W4297241591 modified "2023-09-25" @default.
- W4297241591 title "Modelling urea cycle disorders using iPSCs" @default.
- W4297241591 cites W1566010314 @default.
- W4297241591 cites W1862365802 @default.
- W4297241591 cites W1870080799 @default.
- W4297241591 cites W1879322546 @default.
- W4297241591 cites W1934207236 @default.
- W4297241591 cites W1967223099 @default.
- W4297241591 cites W1971553024 @default.
- W4297241591 cites W1971822008 @default.
- W4297241591 cites W1972021376 @default.
- W4297241591 cites W1977841063 @default.
- W4297241591 cites W1983366316 @default.
- W4297241591 cites W1986543208 @default.
- W4297241591 cites W1990235141 @default.
- W4297241591 cites W1991403924 @default.
- W4297241591 cites W2008485310 @default.
- W4297241591 cites W2010400984 @default.
- W4297241591 cites W2010546163 @default.
- W4297241591 cites W2013232839 @default.
- W4297241591 cites W2017381676 @default.
- W4297241591 cites W2020755558 @default.
- W4297241591 cites W2042768109 @default.
- W4297241591 cites W2045899482 @default.
- W4297241591 cites W2051978340 @default.
- W4297241591 cites W2053755663 @default.
- W4297241591 cites W2057159461 @default.
- W4297241591 cites W2063629303 @default.
- W4297241591 cites W2066858504 @default.
- W4297241591 cites W2068811343 @default.
- W4297241591 cites W2071808643 @default.
- W4297241591 cites W2072624946 @default.
- W4297241591 cites W2082223061 @default.
- W4297241591 cites W2082314612 @default.
- W4297241591 cites W2082356514 @default.
- W4297241591 cites W2085993152 @default.
- W4297241591 cites W2093424805 @default.
- W4297241591 cites W2097746233 @default.
- W4297241591 cites W2108299788 @default.
- W4297241591 cites W2111505666 @default.
- W4297241591 cites W2116507889 @default.
- W4297241591 cites W2116524462 @default.
- W4297241591 cites W2128218526 @default.
- W4297241591 cites W2130093373 @default.
- W4297241591 cites W2131635560 @default.
- W4297241591 cites W2138977668 @default.
- W4297241591 cites W2170491580 @default.
- W4297241591 cites W2170515032 @default.
- W4297241591 cites W2184991014 @default.
- W4297241591 cites W2252568502 @default.
- W4297241591 cites W2314125259 @default.
- W4297241591 cites W2321561488 @default.
- W4297241591 cites W2322149859 @default.
- W4297241591 cites W2324542890 @default.
- W4297241591 cites W2336828812 @default.
- W4297241591 cites W2380796370 @default.
- W4297241591 cites W2465378683 @default.
- W4297241591 cites W2466033010 @default.
- W4297241591 cites W2473248288 @default.
- W4297241591 cites W2517576633 @default.
- W4297241591 cites W2558887711 @default.
- W4297241591 cites W2569831080 @default.
- W4297241591 cites W2581828742 @default.
- W4297241591 cites W2587337751 @default.
- W4297241591 cites W2594256637 @default.
- W4297241591 cites W2596867059 @default.
- W4297241591 cites W2608500349 @default.
- W4297241591 cites W2611134270 @default.
- W4297241591 cites W2611138580 @default.
- W4297241591 cites W2611661371 @default.
- W4297241591 cites W2613056759 @default.
- W4297241591 cites W2619400894 @default.
- W4297241591 cites W2753211361 @default.
- W4297241591 cites W2754108877 @default.
- W4297241591 cites W2773124324 @default.
- W4297241591 cites W2776042747 @default.
- W4297241591 cites W2783420020 @default.
- W4297241591 cites W2787121063 @default.
- W4297241591 cites W2804438136 @default.
- W4297241591 cites W2886709698 @default.
- W4297241591 cites W2887301398 @default.
- W4297241591 cites W2888014775 @default.
- W4297241591 cites W2892080354 @default.
- W4297241591 cites W2902585499 @default.
- W4297241591 cites W2912077551 @default.
- W4297241591 cites W2913945623 @default.
- W4297241591 cites W2921828767 @default.
- W4297241591 cites W2938571175 @default.
- W4297241591 cites W2942799604 @default.
- W4297241591 cites W2946581505 @default.
- W4297241591 cites W2951597236 @default.
- W4297241591 cites W2954087090 @default.
- W4297241591 cites W2955916722 @default.
- W4297241591 cites W2968525218 @default.
- W4297241591 cites W2971511984 @default.