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:…Human induced pluripotent stem cells (iPSCs) as a model for the study of neural development in Parkinson’s Disease patients carrying GBA mutations
Objective: The aim of this study is to evaluate in vitro development of human induced pluripotent stem cells (iPSCs) [1] derived from fibroblasts of Parkinson’s…Allogeneic bone marrow-derived mesenchymal stem cells safety and tolerability in idiopathic Parkinson’s disease
Objective: Prove safety and feasibility of delivering allogeneic bone marrow-derived mesenchymal stem cells (MSC) intravenously in escalated doses to patients with idiopathic Parkinson’s disease (PD).…Distribution of intranasal hNSCs in a mouse model of MPTP-induced Parkinson’s disease
Objective: We aimed to map the distribution of human neural stem cells (hNSCs) in the brain and peripheral tissues after intranasal administration in a mouse…Circadian rhythm alterations in an in vitro cellular model of Spinocerebellar ataxia type 17
Objective: To study in vitro expression of circadian rhythm genes (CLOCK, BMAL) in fibroblasts and neural cells of SCA17 patients. Background: Spinocerebellar Ataxia 17 (SCA17)…Investigating the impact of a-synuclein pathology on human embryonic stem cell derived dopaminergic neurons transplanted in an accelerated a-synuclein rat model of Parkinson’s disease
Objective: In this study, we assess how transplanted human embryonic stem cell (hESC) derived dopaminergic (DA) neurons survive, mature, integrate and innervate the host circuitry…Neuritic pathology in Parkinson’s Disease results from loss of anti-oxidant response activity
Objective: To determine the link between α-syn accumulation and neuritic pathology in Parkinson’s Disease (PD). Background: Neurological dysfunction in PD is highly associated with pathological…Disease-in-a-dish: drug discovery using patient-derived stem cells in Hereditary Spastic Paraplegia
Objective: Identify a drug treatment for Hereditary Spastic Paraplegia (HSP) using patient-derived stem cell models Background: HSP is a neurological disorder characterised by axonal degeneration…Texas Biorepository of Human Induced Pluripotent Stem Cells for Studying Parkinson’s Disease
Objective: To develop a biorepository of human induced pluripotent stem cells for studying Parkinson’s disease(PD). Background: Genetically engineered stem cells called “induced pluripotent stem cells”…