Genome engineering tools for building cellular models of disease

Jennie Lin, Kiran Musunuru*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

14 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)3222-3231
Number of pages10
JournalFEBS Journal
DOIs
StatePublished - Sep 1 2016

Keywords

  • CRISPR/Cas9
  • TALENs
  • genome editing
  • induced pluripotent stem cell
  • zinc finger nucleases

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Biology
  • Cell Biology

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