Breakthrough Moments: Genome Editing and Organoids

Cell Stem Cell. 2019 Jun 6;24(6):841-842. doi: 10.1016/j.stem.2019.05.008.

Abstract

Six years ago, Schwank et al. (2013) adapted CRISPR-Cas9 and organoid technology to repair genetic diseases in patient-derived tissues. We shine a spotlight on how this work has inspired the development of tools to study and correct genetic diseases in experimental systems, with the ultimate goal of treating human disease.

MeSH terms

  • Animals
  • CRISPR-Cas Systems
  • Disease Models, Animal
  • Gene Editing / methods*
  • Genetic Diseases, Inborn / genetics
  • Genetic Diseases, Inborn / pathology*
  • Genetic Therapy / methods*
  • Humans
  • Organoids / pathology*
  • Organoids / physiology