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”…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…MicroRNA expression misregulation in iPSC-derived dopaminergic neurons from sporadic and LRRK2-associated Parkinson disease patients
Objective: To investigate whether miRNA expression alterations occur in iPSC-derived DAn from sporadic PD (sPD) as well as monogenic LRRK2-associated PD patients. Background: MicroRNA (miRNA)…Cell and animal models lacking RAB39B show biochemical and behavioural phenotypes that model aspects of Parkinson’s disease
Objective: To determine the pathogenic mechanisms underlying RAB39B-mediated Parkinson’s disease (PD). Background: PD is a neurodegenerative disorder characterized by loss of dopaminergic neurons in the…Transplantation therapy of human iPS cell-derived dopamine neural progenitor cells for Parkinson’s disease
Objective: To examine the efficacy of midbrain dopaminergic progenitors derived from human iPS cells, as the cell source for Parkinson's disease model mouse, by analyzing…Disease Modeling of DYT1 Using Patient Induced Pluripotent Stem Cells
Objective: To create a resource of induced pluripotent stem cells (iPSCs) as models for exploring mechanisms of pathogenesis in DYT1 dystonia. Background: DYT1 an autosomal…Generation and in-depth characterization of induced pluripotent stem cell (iPSC) lines from 10 affected and unaffected carriers of THAP1 mutations
Objective: To establish induced pluripotent stem cells (iPSCs) of affected and unaffected Mutation carriers to investigate disease mechanisms of THAP1 in dystonia. Background: Mutations in…Disease Modeling of Lesch-Nyhan Disease Using Induced Pluripotent Stem Cells
Objective: To create a resource of induced pluripotent stem cells (iPSCs) as models for exploring mechanisms of pathogenesis in Lesch-Nyhan disease (LND). Background: LND is…CRISPR/Cas9-based fluorescent tyrosine hydroxylase-reporter lines
Objective: To generate and characterize TH-GFP and -mCherry reporter iPSC lines. Background: Human induced pluripotent stem cells (iPSCs)-based dopaminergic neuronal (iDA) cultures emerged as a…
Abstracts from the International Congress of Parkinson’s and Movement Disorders.