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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Asıl Yazarlar: Patrick C Lee (Yazar), Brian Truong (Yazar), Agustin Vega-Crespo (Yazar), W Blake Gilmore (Yazar), Kip Hermann (Yazar), Stephanie AK Angarita (Yazar), Jonathan K Tang (Yazar), Katherine M Chang (Yazar), Austin E Wininger (Yazar), Alex K Lam (Yazar), Benjamen E Schoenberg (Yazar), Stephen D Cederbaum (Yazar), April D Pyle (Yazar), James A Byrne (Yazar), Gerald S Lipshutz (Yazar)
Materyal Türü: Kitap
Baskı/Yayın Bilgisi: Elsevier, 2016-01-01T00:00:00Z.
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