Parkinson’s Progression Markers Initiative (PPMI): Investigating Biomarker Driven PD Progression
Objective: PPMI is a longitudinal, observational study enrolling PD participants, individuals at risk for PD, and healthy controls (HCs). The study investigates clinical, imaging, genetic…The Potential for Rational Immune System Manipulation to Prevent Emergence of Synucleinopathy Manifestations in Persons with REM Sleep Behavior Disorder (RBD)
Objective: To explore the potential for an immunologically based intervention to slow evolution of symptoms in persons with prodromal or Stage 2b (Simuni et al.…The Purely Thermodynamic Anti-prionic Mode of Action for the Treatment of Parkinson’s Disease and other Neurodegenerative Diseases
Objective: Demonstration of the anti-prionic MoA for α-synuclein in vitro, PoC in vivo, as well as ex vivo. Background: Neurodegenerative protein-misfolding diseases, like Alzheimer’s (AD)…TM9SF2 Knockdown Decreases Pathological Alpha-synuclein Aggregation In Vivo
Objective: To determine whether knockdown of transmembrane 9 super family 2 (TM9SF2) decreases the formation of alpha-synuclein inclusions and toxicity to nigral neurons in the…Investigating the pathogenesis of GBA1-associated Parkinson’s disease using GBA1 isogenic iPSC lines
Objective: We aim to explore the pathogenesis of GBA1-associated synucleinopathies and discern the gain- vs. loss-of-function hypotheses by genetically engineering GBA1 in an iPSC line derived…Untargeted Serum Metabolomics Identifies Glycine-Serine Metabolism Disruption as Biochemical Bignature in Parkinson’s Disease
Objective: We attempted to identify a metabolomic signature distinctive of Parkinson’s disease (PD) through an untargeted metabolomics approach. Background: Using an high-performance liquid chromatography targeted…Polyamine pathways: a promising frontier for biomarkers and therapeutic targets in Parkinson’s disease (PD)
Objective: To systematically modulate polyamine (PA) metabolic pathways and investigate PA biomarker findings in PD using Drosophila melanogaster over-expressing human wild-type α-synuclein gene (hSNCA). Our…Development of an AAV-mediated artificial miRNA targeting human alpha-synuclein (α-syn) as a potential gene therapy for Synucleinopathies
Objective: To develop an adeno-associated virus (AAV)-based artificial miRNA (miRNA) system targeting human α-syn. Background: Synucleinopathies such as Parkinson’s disease (PD) and Multiple System Atrophy…Resting-State Network Alterations in Patients with Isolated REM Sleep Behavior Disorder and their Association with Clinical Symptoms
Objective: To characterize brain functional connectivity (FC) in patients with isolated REM sleep behavior disorder (iRBD) and its associations with clinical features. Background: Patients with…Altered activity of lysosomal hydrolases in monocyte-derived macrophages as potential biomarker for differential diagnosis of multiple system atrophy
Objective: The aim was to assess the possibility of differentiating synucleinopathies (Parkinson’s disease (PD), dementia with Lewy bodies (DLB), multiple system atrophy (MSA) by lysosomal…
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