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…Modeling Inflammation in GBA-Associated Parkinson’s Disease: Insights into Disease Mechanisms
Objective: This investigation centers on the role of inflammation in GBA-associated Parkinson’s disease (PD), a neurodegenerative disorder characterized by the progressive degeneration of dopaminergic neurons,…Neuroprotective Effects of ATP6V0C Overexpression in PFF Model Mice
Objective: Elucidating the role of lysosomal dysfunction due to altered stability and expression levels of V-type proton subunit ATP6V0C in the degradation and propagation of…Rescuing alpha-synuclein toxicity through neuron-specific enhancement of autophagy
Objective: In the present study, we aim to counteract regulatory and transcriptional modifiers of autophagy to enhance neuroprotective proteostasis and rescue alpha-synuclein (asyn) toxicity in…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…
- 1
- 2
- 3
- …
- 9
- Next Page »