Exome-wide Burden Analysis Identifies SYT10 as a Genetic Modifier of GBA-PD in UK Biobank
Objective: Identify potential genetic modifiers of GBA1 risk on Parkinson’s disease (PD). Background: GBA1 is a common genetic risk factor for PD. GBA1-carrying PD cases…Impact of rare lysosomal gene variants on Parkinson’s disease
Objective: To investigate the contribution of rare variants in lysosomal genes to Parkinson’s disease (PD) susceptibility. Background: Variants in the lysosomal gene GBA1 are established…Screening for GBA1 p.K198E PD-Risk Variant in Latin America
Objective: We aimed to assess the distribution of the GBA1 p.K198E variant, linked to Gaucher’s disease (GD) and increased Parkinson’s disease (PD) risk, across Latin…CAP-003, a CNS-targeted IV-delivered AAV Gene Therapy Developed for Patients with Parkinson’s Disease associated with GBA1 Mutations
Objective: CAP-003 is a next-generation gene therapy candidate, comprising a novel brain-tropic capsid paired with hGBA1 cargo, that was developed for administration as a single…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:…
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