Restoring Ureagenesis in Hepatocytes by CRISPR/Cas9-mediated Genomic Addition to Arginase-deficient Induced Pluripotent Stem Cells

Urea cycle disorders are incurable enzymopathies that affect nitrogen metabolism and typically lead to hyperammonemia. Arginase deficiency results from a mutation in Arg1, the enzyme regulating the final step of ureagenesis and typically results in developmental disabilities, seizures, spastic diple...

Täydet tiedot

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Bibliografiset tiedot
Julkaisussa:Mol Ther Nucleic Acids
Päätekijät: Lee, Patrick C, Truong, Brian, Vega-Crespo, Agustin, Gilmore, W Blake, Hermann, Kip, Angarita, Stephanie AK, Tang, Jonathan K, Chang, Katherine M, Wininger, Austin E, Lam, Alex K, Schoenberg, Benjamen E, Cederbaum, Stephen D, Pyle, April D, Byrne, James A, Lipshutz, Gerald S
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Nature Publishing Group 2016
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Original Article
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC5155330/
https://ncbi.nlm.nih.gov/pubmed/27898091
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2016.98
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