Delivery technologies for genome editing

Nat Rev Drug Discov. 2017 Jun;16(6):387-399. doi: 10.1038/nrd.2016.280. Epub 2017 Mar 24.

Abstract

With the recent development of CRISPR technology, it is becoming increasingly easy to engineer the genome. Genome-editing systems based on CRISPR, as well as transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), are becoming valuable tools for biomedical research, drug discovery and development, and even gene therapy. However, for each of these systems to effectively enter cells of interest and perform their function, efficient and safe delivery technologies are needed. This Review discusses the principles of biomacromolecule delivery and gene editing, examines recent advances and challenges in non-viral and viral delivery methods, and highlights the status of related clinical trials.

Publication types

  • Review

MeSH terms

  • Animals
  • Biomedical Research / methods
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics
  • Drug Discovery / methods
  • Gene Editing / methods*
  • Gene Transfer Techniques
  • Genetic Engineering / methods*
  • Genetic Therapy / methods*
  • Genome, Human / genetics*
  • Humans
  • Transcription Activator-Like Effector Nucleases / genetics
  • Zinc Fingers / genetics

Substances

  • Transcription Activator-Like Effector Nucleases