title: Design and Validation of CRISPR/Cas9 Systems for Targeted Gene Modification in Induced Pluripotent Stem Cells.
abstract: The CRISPR/Cas9 system is a powerful tool for precision genome editing. The ability to accurately modify genomic DNA in situ with single nucleotide precision opens up new possibilities for not only basic research but also biotechnology applications and clinical translation. In this chapter, we outline the procedures for design, screening, and validation of CRISPR/Cas9 systems for targeted modification of coding sequences in the human genome and how to perform genome editing in induced pluripotent stem cells with high efficiency and specificity.
authors.affiliation: Department of Bioengineering, Rice University, 6500 Main St., Houston, TX, 77030, USA.
authors.forename: Ciaran M
authors.initials: CM
authors.lastname: Lee
authors.affiliation: Department of Bioengineering, Rice University, 6500 Main St., Houston, TX, 77030, USA.
authors.forename: Haibao
authors.initials: H
authors.lastname: Zhu
authors.affiliation: Department of Bioengineering, Rice University, 6500 Main St., Houston, TX, 77030, USA.
authors.forename: Timothy H
authors.initials: TH
authors.lastname: Davis
authors.affiliation: Department of Bioengineering, Rice University, 6500 Main St., Houston, TX, 77030, USA.
authors.forename: Harshahardhan
authors.initials: H
authors.lastname: Deshmukh
authors.affiliation: Department of Bioengineering, Rice University, 6500 Main St., Houston, TX, 77030, USA. gang.bao@rice.edu.
authors.forename: Gang
authors.initials: G
authors.lastname: Bao
mesh_terms: D064113:CRISPR-Cas Systems / Q000235:genetics*; D002460:Cell Line; D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics*; D000072669:Gene Editing / Q000379:methods; D015894:Genome, Human / Q000235:genetics*; D057809:HEK293 Cells; D006801:Humans; D039904:Pluripotent Stem Cells / Q000502:physiology*; D017393:RNA Editing / Q000235:genetics*
keywords: CRISPR; Genome editing; Targeted gene knockout