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Focus: Rare Disease: CRISPR-Mediated Genomic Addition to CPS1 Deficient iPSCs is Insufficient to Restore Nitrogen Homeostasis |
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| Authors: | Matthew Nitzahn,Brian Truong,Suhail Khoja,Agustin Vega-Crespo,Colleen Le,Adam Eliav,Georgios Makris,April D. Pyle,Johannes Hä berle,Gerald S. Lipshutz |
| Abstract: | |
| Keywords: | Carbamoyl phosphate synthetase deficiency hyperammonemia gene therapy CRISPR/Cas9 iPSC |