Cargando…
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...
Autores principales: | , |
---|---|
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 |
_version_ | 1784819459860463616 |
---|---|
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. |
format | Online Article Text |
id | pubmed-9611171 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
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 |
work_keys_str_mv | AT godboutkelly deliveryofrnastospecificorgansbylipidnanoparticlesforgenetherapy AT tremblayjacquesp deliveryofrnastospecificorgansbylipidnanoparticlesforgenetherapy |