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Nanotechnology-based gene therapy as a credible tool in the treatment of Alzheimer’s disease

Toxic aggregated amyloid-β accumulation is a key pathogenic event in Alzheimer’s disease. Treatment approaches have focused on the suppression, deferral, or dispersion of amyloid-β fibers and plaques. Gene therapy has evolved as a potential therapeutic option for treating Alzheimer’s disease, owing...

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Autores principales: Unnisa, Aziz, Greig, Nigel H., Kamal, Mohammad Amjad
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Wolters Kluwer - Medknow 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10328264/
https://www.ncbi.nlm.nih.gov/pubmed/37056119
http://dx.doi.org/10.4103/1673-5374.369096
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author Unnisa, Aziz
Greig, Nigel H.
Kamal, Mohammad Amjad
author_facet Unnisa, Aziz
Greig, Nigel H.
Kamal, Mohammad Amjad
author_sort Unnisa, Aziz
collection PubMed
description Toxic aggregated amyloid-β accumulation is a key pathogenic event in Alzheimer’s disease. Treatment approaches have focused on the suppression, deferral, or dispersion of amyloid-β fibers and plaques. Gene therapy has evolved as a potential therapeutic option for treating Alzheimer’s disease, owing to its rapid advancement over the recent decade. Small interfering ribonucleic acid has recently garnered considerable attention in gene therapy owing to its ability to down-regulate genes with high sequence specificity and an almost limitless number of therapeutic targets, including those that were once considered undruggable. However, lackluster cellular uptake and the destabilization of small interfering ribonucleic acid in its biological environment restrict its therapeutic application, necessitating the development of a vector that can safeguard the genetic material from early destruction within the bloodstream while effectively delivering therapeutic genes across the blood-brain barrier. Nanotechnology has emerged as a possible solution, and several delivery systems utilizing nanoparticles have been shown to bypass key challenges regarding small interfering ribonucleic acid delivery. By reducing the enzymatic breakdown of genetic components, nanomaterials as gene carriers have considerably enhanced the efficiency of gene therapy. Liposomes, polymeric nanoparticles, magnetic nanoparticles, dendrimers, and micelles are examples of nanocarriers that have been designed, and each has its own set of features. Furthermore, recent advances in the specific delivery of neurotrophic compounds via gene therapy have provided promising results in relation to augmenting cognitive abilities. In this paper, we highlight the use of different nanocarriers in targeted gene delivery and small interfering ribonucleic acid-mediated gene silencing as a potential platform for treating Alzheimer’s disease.
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spelling pubmed-103282642023-07-08 Nanotechnology-based gene therapy as a credible tool in the treatment of Alzheimer’s disease Unnisa, Aziz Greig, Nigel H. Kamal, Mohammad Amjad Neural Regen Res Review Toxic aggregated amyloid-β accumulation is a key pathogenic event in Alzheimer’s disease. Treatment approaches have focused on the suppression, deferral, or dispersion of amyloid-β fibers and plaques. Gene therapy has evolved as a potential therapeutic option for treating Alzheimer’s disease, owing to its rapid advancement over the recent decade. Small interfering ribonucleic acid has recently garnered considerable attention in gene therapy owing to its ability to down-regulate genes with high sequence specificity and an almost limitless number of therapeutic targets, including those that were once considered undruggable. However, lackluster cellular uptake and the destabilization of small interfering ribonucleic acid in its biological environment restrict its therapeutic application, necessitating the development of a vector that can safeguard the genetic material from early destruction within the bloodstream while effectively delivering therapeutic genes across the blood-brain barrier. Nanotechnology has emerged as a possible solution, and several delivery systems utilizing nanoparticles have been shown to bypass key challenges regarding small interfering ribonucleic acid delivery. By reducing the enzymatic breakdown of genetic components, nanomaterials as gene carriers have considerably enhanced the efficiency of gene therapy. Liposomes, polymeric nanoparticles, magnetic nanoparticles, dendrimers, and micelles are examples of nanocarriers that have been designed, and each has its own set of features. Furthermore, recent advances in the specific delivery of neurotrophic compounds via gene therapy have provided promising results in relation to augmenting cognitive abilities. In this paper, we highlight the use of different nanocarriers in targeted gene delivery and small interfering ribonucleic acid-mediated gene silencing as a potential platform for treating Alzheimer’s disease. Wolters Kluwer - Medknow 2023-03-03 /pmc/articles/PMC10328264/ /pubmed/37056119 http://dx.doi.org/10.4103/1673-5374.369096 Text en Copyright: © 2023 Neural Regeneration Research https://creativecommons.org/licenses/by-nc-sa/4.0/This is an open access journal, and articles are distributed under the terms of the Creative Commons AttributionNonCommercial-ShareAlike 4.0 License, which allows others to remix, tweak, and build upon the work non-commercially, as long as appropriate credit is given and the new creations are licensed under the identical terms.
spellingShingle Review
Unnisa, Aziz
Greig, Nigel H.
Kamal, Mohammad Amjad
Nanotechnology-based gene therapy as a credible tool in the treatment of Alzheimer’s disease
title Nanotechnology-based gene therapy as a credible tool in the treatment of Alzheimer’s disease
title_full Nanotechnology-based gene therapy as a credible tool in the treatment of Alzheimer’s disease
title_fullStr Nanotechnology-based gene therapy as a credible tool in the treatment of Alzheimer’s disease
title_full_unstemmed Nanotechnology-based gene therapy as a credible tool in the treatment of Alzheimer’s disease
title_short Nanotechnology-based gene therapy as a credible tool in the treatment of Alzheimer’s disease
title_sort nanotechnology-based gene therapy as a credible tool in the treatment of alzheimer’s disease
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10328264/
https://www.ncbi.nlm.nih.gov/pubmed/37056119
http://dx.doi.org/10.4103/1673-5374.369096
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