Current progress in therapeutic gene editing for monogenic diseases

  • Prakash V
  • Moore M
  • Yáñez-Muñoz R
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Abstract

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.

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Authors

  • Versha Prakash

  • Marc Moore

  • Rafael J. Yáñez-Muñoz

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