title: Genome engineering tools for building cellular models of disease.
abstract: With the recent development of methods for genome editing of human pluripotent stem cells, study of the genetic basis of human diseases has been rapidly advancing. Genome-edited differentiated stem cells have provided new and more accurate insights into genomic underpinnings of diseases for which there have not been adequate treatments, and moving toward clinical application of genome editing holds great promise for acceleration of therapeutic translation. Here, we review recent advances in genome-editing technologies and their application to human biology in disease modeling and beyond.
authors.affiliation: Renal, Electrolyte, and Hypertension Division, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: Jennie
authors.initials: J
authors.lastname: Lin
authors.affiliation: Cardiovascular Institute, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.|Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: Kiran
authors.initials: K
authors.lastname: Musunuru
mesh_terms: D000818:Animals; D064113:CRISPR-Cas Systems; D004194:Disease / Q000235:genetics*; D000072669:Gene Editing / Q000379:methods / Q000639:trends; D005818:Genetic Engineering / Q000379:methods* / Q000639:trends; D006801:Humans; D057026:Induced Pluripotent Stem Cells; D008957:Models, Genetic; D000069896:Transcription Activator-Like Effector Nucleases; D016335:Zinc Fingers
chemical_list: D000069896:Transcription Activator-Like Effector Nucleases
keywords: CRISPR/Cas9; TALENs; genome editing; induced pluripotent stem cell; zinc finger nucleases