From Genomics to Gene Therapy: Induced Pluripotent Stem Cells Meet Genome Editing

Annu Rev Genet. 2015;49:47-70. doi: 10.1146/annurev-genet-112414-054926. Epub 2015 Sep 25.


The advent of induced pluripotent stem (iPS) cells has opened up numerous avenues of opportunity for cell therapy, including the initiation in September 2014 of the first human clinical trial to treat dry age-related macular degeneration. In parallel, advances in genome-editing technologies by site-specific nucleases have dramatically improved our ability to edit endogenous genomic sequences at targeted sites of interest. In fact, clinical trials have already begun to implement this technology to control HIV infection. Genome editing in iPS cells is a powerful tool and enables researchers to investigate the intricacies of the human genome in a dish. In the near future, the groundwork laid by such an approach may expand the possibilities of gene therapy for treating congenital disorders. In this review, we summarize the exciting progress being made in the utilization of genomic editing technologies in pluripotent stem cells and discuss remaining challenges toward gene therapy applications.

Keywords: CRISPR-Cas9; TALEN; genetic correction; pluripotent stem cells; programmable nuclease.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems
  • Chromosome Deletion
  • Chromosomes, Artificial, Bacterial
  • Gene Targeting / methods
  • Genetic Engineering / methods*
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Genomics / methods*
  • Humans
  • Induced Pluripotent Stem Cells / physiology*
  • Mice