Neurological manifestations and genotypes of Gaucher disease type 3: MDSGene systematic review
Objective: To characterize the spectrum of neurological manifestations of Gaucher disease type 3 (GD3) and identify the most common GBA1 variants. Background: Gaucher disease is…Modifiers of Parkinson’s disease associated with mutations in the GBA1 gene based on transcriptome data validation
Objective: To identify modifiers of Parkinson's disease (PD) among carriers of mutations in the GBA1 gene based on transcriptome data validation analysis by the assessing…Oligodendrocyte-specific mRNA therapy with lipid nanoparticles
Objective: To develop lipid nanoparticles with high efficiency and specificity to introduce mRNA into oligodendrocytes. To denmonstrate the potential therapy of Krabbe disease as one…G-Can, the GBA1 Canada Initiative
Objective: G-Can, the GBA1 Canada Initiative, is an open-science platform which aims to advance GBA1 research and develop treatments for GBA1 mutation-related Parkinson's disease (PD).…Investigating the cellular consequences of the G2385R LRRK2 variant
Objective: The objective of this study is to characterise the biochemical and cellular impact of the G2385R coding variant in LRRK2, a risk variant for…CSM-101 is a Small Molecule Agonist of TRPML1 for Parkinson’s-Related Disorders
Objective: (i) To develop a brain-penetrant small molecule agonist for the lysosomal TRPML1 channel and, (ii) Perform preclinical studies supporting development in Parkinson’s-related disorders. Background:…VQ-101, a CNS-Penetrant Small Molecule Allosteric Activator of Glucocerebrosidase (GCase), Demonstrates Favorable Tolerability and Sustained Activation of Lysosomal GCase in Individuals with Parkinson’s Disease
Objective: To assess the safety, tolerability, pharmacokinetics and pharmacodynamics of VQ-101 in healthy volunteers (HVs), and individuals with Parkinson’s disease (PD) with and without GBA1…The sphingolipid Lyso-Gb3, a biomarker for the lysosomal storage disorder Fabry Disease, is significantly elevated in blood from patients with Parkinson’s Disease
Objective: To better understand lysosomal dysfunction in Parkinson’s Disease. Background: Lysosomal dysfunction and α-synuclein accumulation play central roles in Parkinson’s Disease. They are interconnected by…The Role of Autophagy Lysosomal and Ubiquitin Proteasomal System in the Pathophysiology of Parkinson’s disease.
Objective: The research how to better understand the molecular mechanism initiating the etiology of Parkinson's disease in order to appropriate the precise diagnosis and therapeutic…GCase and Ganglioside GM1 Interaction in Parkinson’s Disease
Objective: The project aims to further understand the role of the ganglioside GM1 in the relationship between GBA1 mutations and higher α-synuclein levels. Background: Among…
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