Open AccessEfficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes
Author(s) -
Samantha L. Ginn,
Anais K. Amaya,
Sophia H.Y. Liao,
Erhua Zhu,
Sharon C. Cunningham,
Michael Lee,
Claus V. Hallwirth,
Grant J. Logan,
Szun S. Tay,
Anthony J. Cesare,
Hilda A. Pickett,
Markus Grompe,
Kimberley L. Dilworth,
Leszek Lisowski,
Ian E. Alexander
Publication year - 2019
Publication title -
jhep reports
Language(s) - English
Resource type - Journals
ISSN - 2589-5559
DOI - 10.1016/j.jhepr.2019.100065
Subject(s) - genome editing , locus (genetics) , biology , minigene , crispr , gene , in vivo , cas9 , adeno associated virus , guide rna , computational biology , genetics , microbiology and biotechnology , recombinant dna , vector (molecular biology) , messenger rna , alternative splicing
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