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Assessment of Different Niosome Formulations for Optogenetic Applications: Morphological and Electrophysiological Effects
Gene therapy and optogenetics are becoming promising tools for treating several nervous system pathologies. Currently, most of these approaches use viral vectors to transport the genetic material inside the cells, but viruses present some potential risks, such as marked immunogenicity, insertional m...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10384779/ https://www.ncbi.nlm.nih.gov/pubmed/37514046 http://dx.doi.org/10.3390/pharmaceutics15071860 |
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author | Celdrán, José David Humphreys, Lawrence González, Desirée Soto-Sánchez, Cristina Martínez-Navarrete, Gema Maldonado, Iván Gallego, Idoia Villate-Beitia, Ilia Sainz-Ramos, Myriam Puras, Gustavo Pedraz, José Luis Fernández, Eduardo |
author_facet | Celdrán, José David Humphreys, Lawrence González, Desirée Soto-Sánchez, Cristina Martínez-Navarrete, Gema Maldonado, Iván Gallego, Idoia Villate-Beitia, Ilia Sainz-Ramos, Myriam Puras, Gustavo Pedraz, José Luis Fernández, Eduardo |
author_sort | Celdrán, José David |
collection | PubMed |
description | Gene therapy and optogenetics are becoming promising tools for treating several nervous system pathologies. Currently, most of these approaches use viral vectors to transport the genetic material inside the cells, but viruses present some potential risks, such as marked immunogenicity, insertional mutagenesis, and limited insert gene size. In this framework, non-viral nanoparticles, such as niosomes, are emerging as possible alternative tools to deliver genetic material, avoiding the aforementioned problems. To determine their suitability as vectors for optogenetic therapies in this work, we tested three different niosome formulations combined with three optogenetic plasmids in rat cortical neurons in vitro. All niosomes tested successfully expressed optogenetic channels, which were dependent on the ratio of niosome to plasmid, with higher concentrations yielding higher expression rates. However, we found changes in the dendritic morphology and electrophysiological properties of transfected cells, especially when we used higher concentrations of niosomes. Our results highlight the potential use of niosomes for optogenetic applications and suggest that special care must be taken to achieve an optimal balance of niosomes and nucleic acids to achieve the therapeutic effects envisioned by these technologies. |
format | Online Article Text |
id | pubmed-10384779 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-103847792023-07-30 Assessment of Different Niosome Formulations for Optogenetic Applications: Morphological and Electrophysiological Effects Celdrán, José David Humphreys, Lawrence González, Desirée Soto-Sánchez, Cristina Martínez-Navarrete, Gema Maldonado, Iván Gallego, Idoia Villate-Beitia, Ilia Sainz-Ramos, Myriam Puras, Gustavo Pedraz, José Luis Fernández, Eduardo Pharmaceutics Article Gene therapy and optogenetics are becoming promising tools for treating several nervous system pathologies. Currently, most of these approaches use viral vectors to transport the genetic material inside the cells, but viruses present some potential risks, such as marked immunogenicity, insertional mutagenesis, and limited insert gene size. In this framework, non-viral nanoparticles, such as niosomes, are emerging as possible alternative tools to deliver genetic material, avoiding the aforementioned problems. To determine their suitability as vectors for optogenetic therapies in this work, we tested three different niosome formulations combined with three optogenetic plasmids in rat cortical neurons in vitro. All niosomes tested successfully expressed optogenetic channels, which were dependent on the ratio of niosome to plasmid, with higher concentrations yielding higher expression rates. However, we found changes in the dendritic morphology and electrophysiological properties of transfected cells, especially when we used higher concentrations of niosomes. Our results highlight the potential use of niosomes for optogenetic applications and suggest that special care must be taken to achieve an optimal balance of niosomes and nucleic acids to achieve the therapeutic effects envisioned by these technologies. MDPI 2023-07-01 /pmc/articles/PMC10384779/ /pubmed/37514046 http://dx.doi.org/10.3390/pharmaceutics15071860 Text en © 2023 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 | Article Celdrán, José David Humphreys, Lawrence González, Desirée Soto-Sánchez, Cristina Martínez-Navarrete, Gema Maldonado, Iván Gallego, Idoia Villate-Beitia, Ilia Sainz-Ramos, Myriam Puras, Gustavo Pedraz, José Luis Fernández, Eduardo Assessment of Different Niosome Formulations for Optogenetic Applications: Morphological and Electrophysiological Effects |
title | Assessment of Different Niosome Formulations for Optogenetic Applications: Morphological and Electrophysiological Effects |
title_full | Assessment of Different Niosome Formulations for Optogenetic Applications: Morphological and Electrophysiological Effects |
title_fullStr | Assessment of Different Niosome Formulations for Optogenetic Applications: Morphological and Electrophysiological Effects |
title_full_unstemmed | Assessment of Different Niosome Formulations for Optogenetic Applications: Morphological and Electrophysiological Effects |
title_short | Assessment of Different Niosome Formulations for Optogenetic Applications: Morphological and Electrophysiological Effects |
title_sort | assessment of different niosome formulations for optogenetic applications: morphological and electrophysiological effects |
topic | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10384779/ https://www.ncbi.nlm.nih.gov/pubmed/37514046 http://dx.doi.org/10.3390/pharmaceutics15071860 |
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