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

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 promi...

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Detalles Bibliográficos
Autores principales: Godbout, Kelly, Tremblay, Jacques P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9611171/
https://www.ncbi.nlm.nih.gov/pubmed/36297564
http://dx.doi.org/10.3390/pharmaceutics14102129
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author Godbout, Kelly
Tremblay, Jacques P.
author_facet Godbout, Kelly
Tremblay, Jacques P.
author_sort Godbout, Kelly
collection PubMed
description 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.
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spelling pubmed-96111712022-10-28 Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy Godbout, Kelly Tremblay, Jacques P. Pharmaceutics Review 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. MDPI 2022-10-07 /pmc/articles/PMC9611171/ /pubmed/36297564 http://dx.doi.org/10.3390/pharmaceutics14102129 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Godbout, Kelly
Tremblay, Jacques P.
Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy
title Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy
title_full Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy
title_fullStr Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy
title_full_unstemmed Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy
title_short Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy
title_sort delivery of rnas to specific organs by lipid nanoparticles for gene therapy
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9611171/
https://www.ncbi.nlm.nih.gov/pubmed/36297564
http://dx.doi.org/10.3390/pharmaceutics14102129
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