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...

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Main Authors: Patrick C Lee (Author), Brian Truong (Author), Agustin Vega-Crespo (Author), W Blake Gilmore (Author), Kip Hermann (Author), Stephanie AK Angarita (Author), Jonathan K Tang (Author), Katherine M Chang (Author), Austin E Wininger (Author), Alex K Lam (Author), Benjamen E Schoenberg (Author), Stephen D Cederbaum (Author), April D Pyle (Author), James A Byrne (Author), Gerald S Lipshutz (Author)
Format: Book
Published: Elsevier, 2016-01-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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