Progress and challenges towards CRISPR/Cas clinical translation

Daniel Rosenblum, Anna Gutkin, Niels Dammes, Dan Peer

Research output: Contribution to journalReview articlepeer-review

30 Scopus citations


CRISPR/Cas systems (clustered regularly interspaced short palindromic repeats) have emerged as powerful tools to manipulate the genome for both research and therapeutic purposes. However, the clinical use of this system is hindered by multiple challenges, such as the rate of off-target effects, editing efficiency, the efficacy of HDR, immunogenicity, as well as development of efficient and safe delivery vehicles that can carry these compounds. Tremendous efforts are being conducted to overcome these challenges, including the discovery and engineering of more precise and efficacious Cas nucleases. Moreover, in recent years multiple viral and non-viral delivery approaches have been explored for in vivo delivery of CRISPR components. Here, we summarize the available CRISPR/Cas toolbox for genome editing as well as the recently developed in vivo delivery vehicles for CRISPR/Cas system. Furthermore, we discuss the remaining challenges for successful clinical translation of this system and highlight the current clinical applications.

Original languageEnglish (US)
Pages (from-to)176-186
Number of pages11
JournalAdvanced Drug Delivery Reviews
StatePublished - Jan 2020


  • CRISPR/Cas systems
  • Cancer immunotherapy
  • Challenges
  • Clinical translation
  • Delivery
  • Gene therapy
  • Non-viral vectors
  • Viral vectors

ASJC Scopus subject areas

  • Pharmaceutical Science


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