Advanced drug delivery reviewsJun 10, 2024

Using fat-based particles to deliver gene-editing tools for medical treatments

mRNA Technology Weekly Brief ↗PubMed ↗DOI ↗

Updated Mar 18, 2026

Abstract

Lipid nanoparticles (LNPs) are FDA-approved for RNA delivery and may enhance the delivery of CRISPR/Cas technology.

CRISPR/Cas technology shows potential for treating various diseases, but delivery challenges hinder in-vivo gene therapy.
LNPs can encapsulate large mRNA molecules along with single guide RNAs, which could facilitate CRISPR/Cas delivery.
Precise targeting in-vivo remains a significant obstacle, necessitating further optimization of LNP formulations.
Modifications to LNP structures and the incorporation of targeting ligands may improve organ and cell type specificity.
Base and prime editing technologies offer potential for precise genetic modifications without creating harmful double-strand breaks.

Simplified

CRISPR/Cas technology presents a promising approach for treating a wide range of diseases, including cancer and genetic disorders. Despite its potential, the translation of CRISPR/Cas into effective in-vivo gene therapy encounters challenges, primarily due to the need for safe and efficient delivery mechanisms. Lipid nanoparticles (LNPs), FDA-approved for RNA delivery, show potential for delivering also CRISPR/Cas, offering the capability to efficiently encapsulate large mRNA molecules with single guide RNAs. However, achieving precise targeting in-vivo remains a significant obstacle, necessitating further research into optimizing LNP formulations. Strategies to enhance specificity, such as modifying LNP structures and incorporating targeting ligands, are explored to improve organ and cell type targeting. Furthermore, the development of base and prime editing technology presents a potential breakthrough, offering precise modifications without generating double-strand breaks (DSBs). Prime editing, particularly when delivered via targeted LNPs, holds promise for treating diverse diseases safely and precisely. This review assesses both the progress made and the persistent challenges faced in using LNP-encapsulated CRISPR-based technologies for therapeutic purposes, with a particular focus on clinical translation.

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Using fat-based particles to deliver gene-editing tools for medical treatments

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