Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles

Jun 20, 2019Advanced materials (Deerfield Beach, Fla.)

Fast and Efficient Genome Editing Inside the Body Using Special Lipid and RNA Nanoparticles

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Abstract

BAMEA-O16B can achieve up to 90% knockout of green fluorescent protein expression in human embryonic kidney cells within 24 hours of mRNA delivery.

  • BAMEA-O16B is a lipid nanoparticle designed to efficiently deliver Cas9 mRNA and single-guide RNA (sgRNA) into specific cells.
  • The nanoparticle releases RNA in response to the reductive environment inside cells, facilitating rapid genome editing.
  • In mouse models, intravenous injection of the BAMEA-O16B/Cas9 mRNA/sgRNA nanoparticle reduces proprotein convertase subtilisin/kexin type 9 levels in serum to 20% of untreated levels.
  • BAMEA-O16B represents one of the most effective carriers for CRISPR/Cas9 delivery, which could enhance the potential applications of this genome editing technology.

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