基因治疗方法治疗帕金森病中线粒体功能障碍。
Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson's Disease.
机构信息
Institute of Neurogenetics, University of Lübeck, 23562 Lübeck, Germany.
Department of Neurology, University Medical Center Schleswig-Holstein, Campus Lübeck, 23562 Lübeck, Germany.
出版信息
Genes (Basel). 2021 Nov 22;12(11):1840. doi: 10.3390/genes12111840.
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.
背景
线粒体功能障碍已被确定为帕金森病患者疾病发作和进展的病理生理学标志。除了整体出现针对这些患者的基因治疗方法外，这一高度相关的分子概念尚未被定义为基因治疗方法的靶点。
方法
本综述将讨论实验证据，表明线粒体功能障碍是治疗单基因和特发性帕金森病患者的可行治疗靶点。此外，我们将重点关注一般治疗策略和需要克服的关键挑战。
结果
我们目前对帕金森病中线粒体生物学的理解为帕金森病的可行治疗策略开辟了道路。可以从原发性线粒体疾病中获得见解。然而，需要解决线粒体靶向基因治疗的大量知识空白和独特挑战，以提供未来的创新治疗方法。
结论
线粒体靶向基因治疗是改善帕金森病中重要原发性疾病机制的潜在策略。然而，需要进一步的研究来解决线粒体靶向基因治疗的独特设计挑战。
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