Advances in CRISPR/Cas9 Technology for in Vivo Translation

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Cicek Y. A., Luther D. C., Kretzmann J. A., Rotello V. M.

BIOLOGICAL & PHARMACEUTICAL BULLETIN, vol.42, no.3, pp.304-311, 2019 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Review
  • Volume: 42 Issue: 3
  • Publication Date: 2019
  • Doi Number: 10.1248/bpb.b18-00811
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Page Numbers: pp.304-311
  • Keywords: clustered regularly interspaced short palindromic repeat, genomics, therapeutics, in vivo, HOMOLOGY-DIRECTED-REPAIR, MOUSE MODEL, MESSENGER-RNA, GENE DELIVERY, ADENOASSOCIATED VIRUS, NANOPARTICLE DELIVERY, NONVIRAL VECTORS, GENOME, ADENOVIRUS, CAS9
  • Middle East Technical University Affiliated: Yes


Clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9 (Cas9) technology has revolutionized therapeutic gene editing by providing researchers with a new method to study and cure diseases previously considered untreatable. While the full range and power of CRISPR technology for therapeutics is being elucidated through in vitro studies, translation to in vivo studies is slow. To date there is no totally effective delivery strategy to carry CRISPR components to the target site in vivo. The complexity of in vivo delivery is furthered by the number of potential delivery methods, the different forms in which CRISPR can be delivered as a therapeutic, and the disease target and tissue type in question. There are major challenges and limitations to delivery strategies, and it is imperative that future directions are guided by well-conducted studies that consider the full effect these variables have on the eventual outcome. In this review we will discuss the advances of the latest in vivo CRISPR/Cas9 delivery strategies and highlight the challenges yet to be overcome.