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Open AccessFeature PaperReview

The Future of Targeted Gene-Based Treatment Strategies and Biomarkers in Parkinson’s Disease

Laboratory of Neurodegenerative Diseases, Centre for Clinical, Experimental Surgery & Translational Research, Biomedical Research Foundation of the Academy of Athens, 11527 Athens, Greece
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Biomolecules 2020, 10(6), 912; https://doi.org/10.3390/biom10060912
Received: 29 April 2020 / Revised: 2 June 2020 / Accepted: 3 June 2020 / Published: 16 June 2020
Biomarkers and disease-modifying therapies are both urgent unmet medical needs in the treatment of Parkinson’s disease (PD) and must be developed concurrently because of their interdependent relationship: biomarkers for the early detection of disease (i.e., prior to overt neurodegeneration) are necessary in order for patients to receive maximal therapeutic benefit and vice versa; disease-modifying therapies must become available for patients whose potential for disease diagnosis and prognosis can be predicted with biomarkers. This review provides an overview of the milestones achieved to date in the therapeutic strategy development of disease-modifying therapies and biomarkers for PD, with a focus on the most common and advanced genetically linked targets alpha-synuclein (SNCA), leucine-rich repeat kinase-2 (LRRK2) and glucocerebrosidase (GBA1). Furthermore, we discuss the convergence of the different pathways and the importance of patient stratification and how these advances may apply more broadly to idiopathic PD. The heterogeneity of PD poses a challenge for therapeutic and biomarker development, however, the one gene- one target approach has brought us closer than ever before to an unprecedented number of clinical trials and biomarker advancements. View Full-Text
Keywords: alpha-synuclein; LRRK2; glucocerebrosidase; GBA; biomarker; disease-modifying; Parkinson’s disease alpha-synuclein; LRRK2; glucocerebrosidase; GBA; biomarker; disease-modifying; Parkinson’s disease
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MDPI and ACS Style

Polissidis, A.; Petropoulou-Vathi, L.; Nakos-Bimpos, M.; Rideout, H.J. The Future of Targeted Gene-Based Treatment Strategies and Biomarkers in Parkinson’s Disease. Biomolecules 2020, 10, 912.

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