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

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

mRNA Technology Weekly Brief ↗PubMed ↗DOI ↗

Updated Mar 29, 2026

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.

Simplified

A main challenge to broaden the biomedical application of CRISPR/Cas9 (clustered regularly interspaced short palindromic repeat (CRISPR) associated protein 9) genome editing technique is the delivery of Cas9 nuclease and single-guide RNA (sgRNA) into the specific cell and organ. An effective and very fast CRISPR/Cas9 genome editing in vitro and in vivo enabled by bioreducible lipid/Cas9 messenger RNA (mRNA) nanoparticle is reported. BAMEA-O16B, a lipid nanoparticle integrated with disulfide bonds, can efficiently deliver Cas9 mRNA and sgRNA into cells while releasing RNA in response to the reductive intracellular environment for genome editing as fast as 24 h post mRNA delivery. It is demonstrated that the simultaneous delivery of Cas9 mRNA and sgRNA using BAMEA-O16B knocks out green fluorescent protein (GFP) expression of human embryonic kidney cells with efficiency up to 90%. Moreover, the intravenous injection of BAMEA-O16B/Cas9 mRNA/sgRNA nanoparticle effectively accumulates in hepatocytes, and knocks down proprotein convertase subtilisin/kexin type 9 level in mouse serum down to 20% of nontreatment. The leading lipid nanoparticle, BAMEA-O16B, represents one of the most efficient CRISPR/Cas9 delivery nanocarriers reported so far, and it can broaden the therapeutic promise of mRNA and CRISPR/Cas9 technique further.

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Conflict of Interest. The authors declare no conflict of interest.

PubMed

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

Abstract

Full Text

Funding

Competing interests

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Abstract

Full Text

Funding

Competing interests

Related papers

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