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…Plasma pTau181 and pTau217 Detect Amyloid Co-pathology and Have Specific Cut-offs in Lewy Body Disease
Objective: We assess: 1) if the diagnostic accuracy of plasma pTau181 differs from pTau217 in detecting amyloidosis (Aβ) in people with Lewy body disease (LBD)…First-In-Human Single and Multiple Ascending Dose Trial Design of LY3962681, a Novel Intrathecally Delivered siRNA Targeting α-synuclein mRNA for the Treatment of Patients with Parkinson’s Disease
Objective: Development of a Phase 1 clinical trial evaluating LY3962681, a novel, intrathecally delivered siRNA directly targeting α-synucleinmRNA (SNCA) to reduce α-synuclein protein (ASYN), for the treatment of…Alpha-synuclein affects PRMT5-mediated cytoprotective pathway in neuronally differentiated SH-SY5Y cell
Objective: To explore the impact of α-synuclein (αS) on PRMT5. Background: Alpha-synuclein (αS) is recognized as the pivotal molecule in the pathomechanism of Parkinson's disease.…Structural magnetic resonance imaging patterns reflect α-synuclein propagation in a model of Parkinson’s Disease
Objective: To evaluate structural magnetic resonance imaging (MRI) pattern modifications in an α-synuclein model of Parkinson’s disease (PD). Background: As an early driver of α-synucleinopathies’…A multicenter, matched case-control study of alpha-synuclein accumulation in the gastrointestinal tract of Parkinson’s disease
Objective: 1) To determine the possibility as a pathologic biomarker of alpha-synuclein (AS) accumulation in the gastrointestinal (GI) tract in patients with Parkinson’s disease (PD) and…Small Molecule Inhibitors for Precise Inhibition of α-Synuclein Oligomer Generation in Parkinson’s Disease (PD)
Objective: To develop small molecule therapeutics for PD that inhibit production of toxic α-synuclein oligomers and reduce aggregate formation, targeting the molecular mechanisms at the…Recruiting people at risk of Parkinson’s disease: comparative data from PPMI Online and HeBA Kassel
Objective: To compare a US- and Germany-based staged screening process to identify people at risk of developing Parkinson’s disease (PD). Background: The Parkinson’s Progression Marker…Longitudinal CSF measurements of alpha-synuclein, amyloid beta and tau in Parkinson disease
Objective: To evaluate whether cerebrospinal fluid (CSF) levels of alpha synuclein (Asyn), amyloid beta (Aβ) and tau change with disease progression and to evaluate whether…Alzheimer’s Disease Co-pathology and its Influence on Biofluid Biomarkers in Autopsied Subjects from the PPMI Study
Objective: To examine the relationship between biofluid biomarkers and post-mortem pathological findings in autopsied subjects from the Parkinson’s Progression Marker Initiative (PPMI). Background: The PPMI…
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