IPSC-derived Cortical Neurons from Parkinson’s Patients with VPS35 Mutation show Defects in Synapto- and Neurogenesis
Objective: Investigate whether the Parkinson’s disease (PD) VPS35 D620N mutation leads to synaptic dysfunction in human cortical iPSC-derived neurons. Background: Early presynaptic dysfunction and cortico-striatal…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…Investigating glutamate toxicity associated to Park2 mutations in pre-clinical models of Parkinson’s Disease
Objective: To generate and characterize induced pluripotent stem cells (iPSCs)-derived in vitro cellular models of Autosomal Recessive Juvenile Parkinsonism (ARJP) carrying Park2 mutations with a…Dopaminergic neuronal cell therapy for Parkinson’s Disease: results from a phase 1 study of Bemdaneprocel
Objective: This first-in-human Phase 1 study aims to assess the safety, tolerability, clinical efficacy, and functional imaging measures of bemdaneprocel in subjects with PD. Background:…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…Functional validation of a mitochondria-specific polygenic risk score in patient-based models for stratification of idiopathic Parkinson’s disease
Objective: The overall objective of this project is to gain essential knowledge on the contribution of genetic variability in nuclear-encoded mitochondrial proteins to idiopathic Parkinson’s…Protein aggregation and calcium dysregulation are the earliest hallmarks of synucleinopathy in enriched iPSC-derived human midbrain dopaminergic neurons
Objective: Develop and adapt a differentiation paradigm to produce enriched hiPSC-derived midbrain dopaminergic neurons (mDA), to resolve the temporal sequence of pathophysiological events in synucleinopathies.…Baseline and six months change in cortical perfusion in a mesenchymal stem cell trial for Parkinson’s disease
Objective: Quantify cortical perfusion at baseline and six months after a single infusion of allogeneic bone marrow-derived mesenchymal stem cells (allo-hMSC) in relation to demographic…Cellular Modeling of Tyrosine Hydroxylase Deficiency Recapitulates Patient Phenotypes and Response to Treatment.
Objective: Characterize a new cellular model of Tyrosine Hydroxylase deficiency (THD) based on induced pluripotent stem cells (iPSCs) to better understand the pathophysiology and test…Tissue-engineered nigrostriatal pathway to rebuild dopaminergic axons in Parkinson’s disease based on clinical design criteria
Objective: To create a tissue-engineered construct with the dopaminergic (DA) cell numbers, axon lengths, and functionality required to reconstruct the human nigrostriatal pathway (NSP). Background:…
Abstracts from the International Congress of Parkinson’s and Movement Disorders.