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Gene-Based Therapeutics for Parkinson’s Disease

Parkinson’s disease (PD) is a complex multifactorial disorder that is not yet fully surmised, and it is only when such a disease is tackled on multiple levels simultaneously that we should expect to see fruitful results. Gene therapy is a modern medical practice that theoretically and, so far, pract...

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Detalles Bibliográficos
Autores principales: Shalaby, Karim E., El-Agnaf, Omar M. A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9331241/
https://www.ncbi.nlm.nih.gov/pubmed/35892690
http://dx.doi.org/10.3390/biomedicines10081790
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author Shalaby, Karim E.
El-Agnaf, Omar M. A.
author_facet Shalaby, Karim E.
El-Agnaf, Omar M. A.
author_sort Shalaby, Karim E.
collection PubMed
description Parkinson’s disease (PD) is a complex multifactorial disorder that is not yet fully surmised, and it is only when such a disease is tackled on multiple levels simultaneously that we should expect to see fruitful results. Gene therapy is a modern medical practice that theoretically and, so far, practically, has demonstrated its capability in joining the battle against PD and other complex disorders on most if not all fronts. This review discusses how gene therapy can efficiently replace current forms of therapy such as drugs, personalized medicine or invasive surgery. Furthermore, we discuss the importance of enhancing delivery techniques to increase the level of transduction and control of gene expression or tissue specificity. Importantly, the results of current trials establish the safety, efficacy and applicability of gene therapy for PD. Gene therapy’s variety of potential in interfering with PD’s pathology by improving basal ganglial circuitry, enhancing dopamine synthesis, delivering neuroprotection or preventing neurodegeneration may one day achieve symptomatic benefit, disease modification and eradication.
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spelling pubmed-93312412022-07-29 Gene-Based Therapeutics for Parkinson’s Disease Shalaby, Karim E. El-Agnaf, Omar M. A. Biomedicines Review Parkinson’s disease (PD) is a complex multifactorial disorder that is not yet fully surmised, and it is only when such a disease is tackled on multiple levels simultaneously that we should expect to see fruitful results. Gene therapy is a modern medical practice that theoretically and, so far, practically, has demonstrated its capability in joining the battle against PD and other complex disorders on most if not all fronts. This review discusses how gene therapy can efficiently replace current forms of therapy such as drugs, personalized medicine or invasive surgery. Furthermore, we discuss the importance of enhancing delivery techniques to increase the level of transduction and control of gene expression or tissue specificity. Importantly, the results of current trials establish the safety, efficacy and applicability of gene therapy for PD. Gene therapy’s variety of potential in interfering with PD’s pathology by improving basal ganglial circuitry, enhancing dopamine synthesis, delivering neuroprotection or preventing neurodegeneration may one day achieve symptomatic benefit, disease modification and eradication. MDPI 2022-07-26 /pmc/articles/PMC9331241/ /pubmed/35892690 http://dx.doi.org/10.3390/biomedicines10081790 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
Shalaby, Karim E.
El-Agnaf, Omar M. A.
Gene-Based Therapeutics for Parkinson’s Disease
title Gene-Based Therapeutics for Parkinson’s Disease
title_full Gene-Based Therapeutics for Parkinson’s Disease
title_fullStr Gene-Based Therapeutics for Parkinson’s Disease
title_full_unstemmed Gene-Based Therapeutics for Parkinson’s Disease
title_short Gene-Based Therapeutics for Parkinson’s Disease
title_sort gene-based therapeutics for parkinson’s disease
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9331241/
https://www.ncbi.nlm.nih.gov/pubmed/35892690
http://dx.doi.org/10.3390/biomedicines10081790
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