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Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics

Abstract

The recent progress in harnessing the efficient and precise method of DNA editing provided by CRISPR/Cas9 is one of the most promising major advances in the field of gene therapy. However, the development of safe and optimally efficient delivery systems for CRISPR/Cas9 elements capable of achieving specific targeting of gene therapy to the location of interest without off-target effects is a primary challenge for clinical therapeutics. Nanoparticles (NPs) provide a promising means to meet such challenges. In this review, we present the most recent advances in developing innovative NP-based delivery systems that efficiently deliver CRISPR/Cas9 constructs and maximize their effectiveness.

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Funding

B. E. G. acknowledges fellowship support from the Alfred P. Sloan Foundation, the University of Iowa Graduate College, and the National GEM Consortium. E. J. Devor acknowledges support from the University of Iowa Department of Obstetrics and Gynecology Research Development Fund. A.K.S. acknowledges support from the National Cancer Institute at the National Institutes of Health (5P30CA086862) and the Lyle and Sharon Bighley Chair of Pharmaceutical Sciences.

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Givens, B.E., Naguib, Y.W., Geary, S.M. et al. Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics. AAPS J 20, 108 (2018). https://doi.org/10.1208/s12248-018-0267-9

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KEY WORDS

  • CRISPR/Cas9
  • gene editing
  • gene delivery
  • nanoparticle