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Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications

Advances in gene therapy have been foreshadowing its potential for the treatment of a vast range of diseases involving genetic malfunctioning. However, its therapeutic efficiency and successful outcome are highly dependent on the development of the ideal gene delivery system. On that matter, silica-...

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Detalles Bibliográficos
Autores principales: Carvalho, Ana Maria, Cordeiro, Rosemeyre A., Faneca, Henrique
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7407166/
https://www.ncbi.nlm.nih.gov/pubmed/32660110
http://dx.doi.org/10.3390/pharmaceutics12070649
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author Carvalho, Ana Maria
Cordeiro, Rosemeyre A.
Faneca, Henrique
author_facet Carvalho, Ana Maria
Cordeiro, Rosemeyre A.
Faneca, Henrique
author_sort Carvalho, Ana Maria
collection PubMed
description Advances in gene therapy have been foreshadowing its potential for the treatment of a vast range of diseases involving genetic malfunctioning. However, its therapeutic efficiency and successful outcome are highly dependent on the development of the ideal gene delivery system. On that matter, silica-based vectors have diverted some attention from viral and other types of non-viral vectors due to their increased safety, easily modifiable structure and surface, high stability, and cost-effectiveness. The versatility of silane chemistry and the combination of silica with other materials, such as polymers, lipids, or inorganic particles, has resulted in the development of carriers with great loading capacities, ability to effectively protect and bind genetic material, targeted delivery, and stimuli-responsive release of cargos. Promising results have been obtained both in vitro and in vivo using these nanosystems as multifunctional platforms in different potential therapeutic areas, such as cancer or brain therapies, sometimes combined with imaging functions. Herein, the current advances in silica-based systems designed for gene therapy are reviewed, including their main properties, fabrication methods, surface modifications, and potential therapeutic applications.
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spelling pubmed-74071662020-08-11 Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications Carvalho, Ana Maria Cordeiro, Rosemeyre A. Faneca, Henrique Pharmaceutics Review Advances in gene therapy have been foreshadowing its potential for the treatment of a vast range of diseases involving genetic malfunctioning. However, its therapeutic efficiency and successful outcome are highly dependent on the development of the ideal gene delivery system. On that matter, silica-based vectors have diverted some attention from viral and other types of non-viral vectors due to their increased safety, easily modifiable structure and surface, high stability, and cost-effectiveness. The versatility of silane chemistry and the combination of silica with other materials, such as polymers, lipids, or inorganic particles, has resulted in the development of carriers with great loading capacities, ability to effectively protect and bind genetic material, targeted delivery, and stimuli-responsive release of cargos. Promising results have been obtained both in vitro and in vivo using these nanosystems as multifunctional platforms in different potential therapeutic areas, such as cancer or brain therapies, sometimes combined with imaging functions. Herein, the current advances in silica-based systems designed for gene therapy are reviewed, including their main properties, fabrication methods, surface modifications, and potential therapeutic applications. MDPI 2020-07-09 /pmc/articles/PMC7407166/ /pubmed/32660110 http://dx.doi.org/10.3390/pharmaceutics12070649 Text en © 2020 by the authors. 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 (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Carvalho, Ana Maria
Cordeiro, Rosemeyre A.
Faneca, Henrique
Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications
title Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications
title_full Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications
title_fullStr Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications
title_full_unstemmed Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications
title_short Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications
title_sort silica-based gene delivery systems: from design to therapeutic applications
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7407166/
https://www.ncbi.nlm.nih.gov/pubmed/32660110
http://dx.doi.org/10.3390/pharmaceutics12070649
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