Design and validation of CRISPR/cas9 systems for targeted gene modification in induced pluripotent stem cells

  • Ciaran M. Lee
  • , Haibao Zhu
  • , Timothy H. Davis
  • , Harshahardhan Deshmukh
  • , Gang Bao

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish
Pages (from-to)3-21
Number of pages19
JournalMethods in Molecular Biology
Volume1498
DOIs
Publication statusPublished - 2017
Externally publishedYes

Keywords

  • CRISPR
  • Genome editing
  • Targeted gene knockout

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