Current Progress in Therapeutic Gene Editing for Monogenic Diseases

Mol Ther. 2016 Mar;24(3):465-74. doi: 10.1038/mt.2016.5. Epub 2016 Jan 14.


Programmable nucleases allow defined alterations in the genome with ease-of-use, efficiency, and specificity. Their availability has led to accurate and widespread genome engineering, with multiple applications in basic research, biotechnology, and therapy. With regard to human gene therapy, nuclease-based gene editing has facilitated development of a broad range of therapeutic strategies based on both nonhomologous end joining and homology-dependent repair. This review discusses current progress in nuclease-based therapeutic applications for a subset of inherited monogenic diseases including cystic fibrosis, Duchenne muscular dystrophy, diseases of the bone marrow, and hemophilia and highlights associated challenges and future prospects.

Publication types

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

MeSH terms

  • Animals
  • Disease Models, Animal
  • Drug Evaluation, Preclinical
  • Gene Editing* / methods
  • Gene Targeting
  • Gene Transfer Techniques
  • Genetic Diseases, Inborn / genetics*
  • Genetic Diseases, Inborn / therapy*
  • Genetic Therapy* / methods
  • Humans
  • Translational Research, Biomedical