Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease

Background: Mitochondrial dysfunction has been identified as a pathophysiological hallmark of disease onset and progression in patients with Parkinsonian disorders. Besides the overall emergence of gene therapies in treating these patients, this highly relevant molecular concept has not yet been def...

Descripción completa

Guardado en:

Detalles Bibliográficos
Autores principales:	Jannik Prasuhn, Norbert Brüggemann
Formato:	article
Lenguaje:	EN
Publicado:	MDPI AG 2021
Materias:	Parkinson’s disease gene therapy mitochondria genome editing Genetics QH426-470
Acceso en línea:	https://doaj.org/article/cf8e89f6e60a4c4384145660bf062bf6
Etiquetas:	Agregar Etiqueta Sin Etiquetas, Sea el primero en etiquetar este registro!

id	oai:doaj.org-article:cf8e89f6e60a4c4384145660bf062bf6
record_format	dspace
spelling	oai:doaj.org-article:cf8e89f6e60a4c4384145660bf062bf62021-11-25T17:42:35ZGene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease10.3390/genes121118402073-4425https://doaj.org/article/cf8e89f6e60a4c4384145660bf062bf62021-11-01T00:00:00Zhttps://www.mdpi.com/2073-4425/12/11/1840https://doaj.org/toc/2073-4425Background: Mitochondrial dysfunction has been identified as a pathophysiological hallmark of disease onset and progression in patients with Parkinsonian disorders. Besides the overall emergence of gene therapies in treating these patients, this highly relevant molecular concept has not yet been defined as a target for gene therapeutic approaches. Methods: This narrative review will discuss the experimental evidence suggesting mitochondrial dysfunction as a viable treatment target in patients with monogenic and idiopathic Parkinson’s disease. In addition, we will focus on general treatment strategies and crucial challenges which need to be overcome. Results: Our current understanding of mitochondrial biology in parkinsonian disorders opens up the avenue for viable treatment strategies in Parkinsonian disorders. Insights can be obtained from primary mitochondrial diseases. However, substantial knowledge gaps and unique challenges of mitochondria-targeted gene therapies need to be addressed to provide innovative treatments in the future. Conclusions: Mitochondria-targeted gene therapies are a potential strategy to improve an important primary disease mechanism in Parkinsonian disorders. However, further studies are needed to address the unique design challenges for mitochondria-targeted gene therapies.Jannik PrasuhnNorbert BrüggemannMDPI AGarticleParkinson’s diseasegene therapymitochondriagenome editingGeneticsQH426-470ENGenes, Vol 12, Iss 1840, p 1840 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Parkinson’s disease gene therapy mitochondria genome editing Genetics QH426-470
spellingShingle	Parkinson’s disease gene therapy mitochondria genome editing Genetics QH426-470 Jannik Prasuhn Norbert Brüggemann Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease
description	Background: Mitochondrial dysfunction has been identified as a pathophysiological hallmark of disease onset and progression in patients with Parkinsonian disorders. Besides the overall emergence of gene therapies in treating these patients, this highly relevant molecular concept has not yet been defined as a target for gene therapeutic approaches. Methods: This narrative review will discuss the experimental evidence suggesting mitochondrial dysfunction as a viable treatment target in patients with monogenic and idiopathic Parkinson’s disease. In addition, we will focus on general treatment strategies and crucial challenges which need to be overcome. Results: Our current understanding of mitochondrial biology in parkinsonian disorders opens up the avenue for viable treatment strategies in Parkinsonian disorders. Insights can be obtained from primary mitochondrial diseases. However, substantial knowledge gaps and unique challenges of mitochondria-targeted gene therapies need to be addressed to provide innovative treatments in the future. Conclusions: Mitochondria-targeted gene therapies are a potential strategy to improve an important primary disease mechanism in Parkinsonian disorders. However, further studies are needed to address the unique design challenges for mitochondria-targeted gene therapies.
format	article
author	Jannik Prasuhn Norbert Brüggemann
author_facet	Jannik Prasuhn Norbert Brüggemann
author_sort	Jannik Prasuhn
title	Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease
title_short	Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease
title_full	Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease
title_fullStr	Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease
title_full_unstemmed	Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease
title_sort	gene therapeutic approaches for the treatment of mitochondrial dysfunction in parkinson’s disease
publisher	MDPI AG
publishDate	2021
url	https://doaj.org/article/cf8e89f6e60a4c4384145660bf062bf6
work_keys_str_mv	AT jannikprasuhn genetherapeuticapproachesforthetreatmentofmitochondrialdysfunctioninparkinsonsdisease AT norbertbruggemann genetherapeuticapproachesforthetreatmentofmitochondrialdysfunctioninparkinsonsdisease
_version_	1718412132642455552

Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease

Ejemplares similares