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Mapping the Diverse and Inclusive Future of Parkinson’s Disease Genetics and Its Widespread Impact
Review

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

by 1,2,3 and 1,2,3,*
1
Institute of Neurogenetics, University of Lübeck, 23562 Lübeck, Germany
2
Department of Neurology, University Medical Center Schleswig-Holstein, Campus Lübeck, 23562 Lübeck, Germany
3
Center for Brain, Behavior and Metabolism, University of Lübeck, 23562 Lübeck, Germany
*
Author to whom correspondence should be addressed.
Academic Editors: Suzanne Lesage and Joanne Trinh
Genes 2021, 12(11), 1840; https://doi.org/10.3390/genes12111840
Received: 30 October 2021 / Revised: 18 November 2021 / Accepted: 19 November 2021 / Published: 22 November 2021
(This article belongs to the Special Issue Parkinson's Disease: Genetics and Pathogenesis)
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. View Full-Text
Keywords: Parkinson’s disease; gene therapy; mitochondria; genome editing Parkinson’s disease; gene therapy; mitochondria; genome editing
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MDPI and ACS Style

Prasuhn, J.; Brüggemann, N. Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease. Genes 2021, 12, 1840. https://doi.org/10.3390/genes12111840

AMA Style

Prasuhn J, Brüggemann N. Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease. Genes. 2021; 12(11):1840. https://doi.org/10.3390/genes12111840

Chicago/Turabian Style

Prasuhn, Jannik, and Norbert Brüggemann. 2021. "Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease" Genes 12, no. 11: 1840. https://doi.org/10.3390/genes12111840

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