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

• Автори: Patrick C Lee (Автор), Brian Truong (Автор), Agustin Vega-Crespo (Автор), W Blake Gilmore (Автор), Kip Hermann (Автор), Stephanie AK Angarita (Автор), Jonathan K Tang (Автор), Katherine M Chang (Автор), Austin E Wininger (Автор), Alex K Lam (Автор), Benjamen E Schoenberg (Автор), Stephen D Cederbaum (Автор), April D Pyle (Автор), James A Byrne (Автор), Gerald S Lipshutz (Автор)
• Формат: Книга
• Опубліковано: Elsevier, 2016-01-01T00:00:00Z.
• Предмети: article
• Онлайн доступ: Connect to this object online.

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...