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)…Expression analysis of candidate genes in a cell model of affected and unaffected THAP1 mutation carriers and controls
Objective: To evaluate the role of expressional changes in carriers of a THAP1 mutation in relation to penetrance of dystonia. Background: THAP1 encodes a transcription…Safety of Bone marrow-derived Allogeneic Mesenchymal Stem Cells in Parkinson’s disease Patients
Objective: Prove safety and tolerability of allogeneic mesenchymal stem cells (MSC) purified from bone marrow derived from a healthy adult and delivered intravenously in escalated…Dissecting the genetic complexity of Parkinson’s disease: a Parkin-GBA study
Objective: In this study, we investigate how the PD risk factor GBA would affect the cellular phenotypes associated with homozygous mutations in Parkin, causing an…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…
Abstracts from the International Congress of Parkinson’s and Movement Disorders.