Programmed Self-Assembly of an Active P22-Cas9 Nanocarrier System

Mol Pharm. 2016 Mar 7;13(3):1191-6. doi: 10.1021/acs.molpharmaceut.5b00822. Epub 2016 Feb 22.

Abstract

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) RNA-guided endonucleases are powerful new tools for targeted genome engineering. These nucleases provide an efficient and precise method for manipulating eukaryotic genomes; however, delivery of these reagents to specific cell-types remains challenging. Virus-like particles (VLPs) derived from bacteriophage P22, are robust supramolecular protein cage structures with demonstrated utility for cell type-specific delivery of encapsulated cargos. Here, we genetically fuse Cas9 to a truncated form of the P22 scaffold protein, which acts as a template for capsid assembly as well as a specific encapsulation signal for Cas9. Our results indicate that Cas9 and a single-guide RNA are packaged inside the P22 VLP, and activity assays indicate that this RNA-guided endonuclease is functional for sequence-specific cleavage of dsDNA targets. This work demonstrates the potential for developing P22 as a delivery vehicle for cell specific targeting of Cas9.

Keywords: Cas9; P22 bacteriophage; VLP; delivery vehicle; gene therapy; genome editing; virus-like particles.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Bacterial Proteins / metabolism*
  • Bacteriophage P22 / metabolism*
  • CRISPR-Associated Protein 9
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics*
  • Drug Carriers / chemistry*
  • Drug Delivery Systems*
  • Endonucleases / genetics
  • Endonucleases / metabolism*
  • Genetic Engineering / methods*
  • Humans
  • Nanoparticles / chemistry*
  • RNA Editing / genetics

Substances

  • Bacterial Proteins
  • Drug Carriers
  • CRISPR-Associated Protein 9
  • Cas9 endonuclease Streptococcus pyogenes
  • Endonucleases