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

Molecular Therapy Nucleic Acids

Volumn 5, Issue , 2016, Pages e394-

  Lee, Patrick C ^a   Truong, Brian ^a   Vega Crespo, Agustin ^a   Gilmore, W Blake ^a   Hermann, Kip ^a   Angarita, Stephanie AK ^a   Tang, Jonathan K ^a   Chang, Katherine M ^a   Wininger, Austin E ^a   Lam, Alex K ^a   Schoenberg, Benjamen E ^a   Cederbaum, Stephen D ^a,b,c   Pyle, April D ^a   Byrne, James A ^a   Lipshutz, Gerald S ^a,b,c  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

arginase; genomic addition; hepatocytes; PSCs; urea cycle

Indexed keywords

ARGINASE; COMPLEMENTARY DNA; HYPOXANTHINE PHOSPHORIBOSYLTRANSFERASE; UREA;

ADULT; ARTICLE; CHILD; CONTROLLED STUDY; CRISPR CAS SYSTEM; DNA MODIFICATION; ENZYME ACTIVITY; EXON; FEMALE; GENE EDITING; GENE EXPRESSION; GENE FUNCTION; GENE LOCUS; HUMAN; HUMAN CELL; INDUCED PLURIPOTENT STEM CELL; LIVER CELL; MALE; MIDDLE AGED; PRESCHOOL CHILD; PRIORITY JOURNAL; UREA CYCLE; YOUNG ADULT; CRISPR CAS9 SYSTEM; HYPERARGININEMIA;

EID: 85015157882     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2016.98     Document Type: Article

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