PARKIN gene replacement by AAV-mediated gene delivery as a therapeutic modality for PARKIN-deficient early-onset familial Parkinson’s disease
Objective: Evaluate PARKIN gene replacement by adeno-associated virus (AAV) vector as a therapeutic modality in relevant disease models of early-onset Parkinson’s Disease (EOPD). Background: Biallelic…Mapping the distribution of neurotransmitters to resting-state functional connectivity in patients with Parkinson’s disease
Objective: To examine the associations between functional connectivity (FC), as measured by resting-state functional magnetic resonance imaging (rs-fMRI), and the spatial distribution of dopamine and…Slc2a13, a risk gene for Parkinson’s disease associated with Snca and Lrrk2
Objective: To exploit the potential function of the risk gene Slc2a13 in PD progression. Background: 越来越多的证据证实了总体遗传位点和候选基因对帕金森病的贡献,以及两个最确定的风险基因,即α-突触核蛋白(Snca)和富含亮氨酸的重复激酶2(Lrrk2)。Slc2a13 编码哺乳动物肌醇转运蛋白,被认为是一种群体依赖性风险基因。越来越多的证据支持这样一种观点,即 Slc2a13 活性的改变强调了 PD 的发展。剖析转运蛋白活性降低如何导致多巴胺能神经元变性和丧失,可能会进一步加深我们对功能的理解。 Method: A total of 338 cases…The impact on religiosity following Deep Brain Stimulation (DBS) in patients with Essential Tremor (ET) and Parkinson’s Disease (PD)
Objective: To determine the impact of DBS on religiosity. Background: Religiosity has been mapped to various brain networks1, 2, and transcranial magnetic stimulation has transiently…Membrane-associated properties and ER stress alterations in LRRK2 I1371V patient iPSC derived neurons increase susceptibility to oxidative stress.
Objective: To evaluate the pathophysiology of the midbrain dopaminergic neurons (mDAN) derived from LRRK2 I1371V PD patient-iPSCs leading to susceptibility to oxidative stress. Background: Studies…GT-02287, a brain-penetrant structurally targeted allosteric regulator for glucocerebrosidase shows evidence of pharmacological efficacy in an animal model of Parkinson’s disease.
Objective: To investigate the effect of the structurally targeted allosteric regulator GT-02287 on rotenone-induced neurotoxic effects in a Parkinson’s disease animal model. Background: Mutations in…PASADENA 1-year open-label extension
Objective: To describe the results of PASADENA 1-year open-label extension (OLE). Background: Prasinezumab is a humanized monoclonal antibody that binds aggregated alpha-synuclein with the potential…A Study to Evaluate the Efficacy and Safety of Intravenous Prasinezumab in Participants With Early Parkinson’s Disease (PADOVA): Rationale, Design, and Baseline Data
Objective: The study will evaluate the efficacy and safety of prasinezumab in individuals with early-stage Parkinson's disease (PD) on stable symptomatic therapy. Background: Available treatments…Glycated alpha-synuclein and it emerging role in early onset of Parkinson’s disease
Objective: To elucidate, the molecular mechanism underlying glycated α-Syn mediated dopaminergic (DAergic) neurodegeneration in the substantia nigra (SN) via the AGE/RAGE signaling pathway. Background: Intracytoplasmic…Impairment of SHH signaling in PD LRRK2 I1371V iPSC derived floor plate cells contribute to ontogenic origin of lower dopaminergic-neuron yield
Objective: To estimate SHH responsiveness & signaling of Floor Plate Cells (FPCs) derived from LRRK2 I1371V PD patient-iPSCs along with its link with LRRK2 I1371V…
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