2. Academic Validation
  3. Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy

Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy

  • Pharmaceutics. 2022 Oct 7;14(10):2129. doi: 10.3390/pharmaceutics14102129.
Kelly Godbout 1 2,  Jacques P Tremblay 1 2
Affiliations

Affiliations

  • 1 Centre de Recherche du CHU de Québec, Laval University, Quebec, QC G1V 4G2, Canada.
  • 2 Department of Molecular Medicine, Faculty of Medicine, Laval University, Quebec, QC G1V 0A6, Canada.
PMID: 36297564 DOI: 10.3390/pharmaceutics14102129
Abstract

Gene therapy holds great promise in the treatment of genetic diseases. It is now possible to make DNA modifications using the CRISPR system. However, a major problem remains: the delivery of these CRISPR-derived technologies to specific organs. Lipid nanoparticles (LNPs) have emerged as a very promising delivery method. However, when delivering LNPs intravenously, most of the cargo is trapped by the liver. Alternatively, injecting them directly into organs, such as the brain, requires more invasive procedures. Therefore, developing more specific LNPs is crucial for their future clinical use. Modifying the composition of the lipids in the LNPs allows more specific deliveries of the LNPs to some organs. In this review, we have identified the most effective compositions and proportions of lipids for LNPs to target specific organs, such as the brain, lungs, muscles, heart, liver, spleen, and bones.

Keywords

CRISPR/Cas9 delivery; gene therapy; lipid nanoparticles; mRNA delivery; specific organ delivery.

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