Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
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Showing 1 to 5 of 5 for “"iPSC models"”.
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Systems Biology Approaches for Elucidating Early ALS Disease Processes
… patient-derived induced pluripotent stem cell (iPSC) models. I present analyses aimed at discovering disease pathways and genes associated with ALS using systems biology approaches. In patients with the C9orf72 mutation, I use a network-based algorithm to identify disrupted pathways enriched for …
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AGGREGATION MECHANISMS OF TDP-43 PROTEIN IN RESPONSE TO STRESS IN AMYOTROPHIC LATERAL SCLEROSIS AND THERAPEUTIC APPROACHES
… affected neurons. The scarcity of experimental models of TDP-43 proteinopathy capable of recapitulating both gain and loss of TDP-43 function has hindered the discovery of novel therapeutic approaches for the treatment of ALS. Aim of this work was therefore to generate human 2D and 3D in vitro …
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Deconvoluting genetic variants associated with aortic disease using stem cell models
… aimed to utilise induced pluripotent stem cell (iPSC) models of TAD to analyse genetic variants from the 100,000 Genomes Project by determining causality, disease mechanisms and identifying drug targets. We focused on three Tier One variants, protein truncating in known aortopathy-related genes. …
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Investigating Parkinson’s Disease human iPSC-derived models through single-cell gene expression
… use in research, induced pluripotent stem cell(iPSC) technologies are still under active development for clinical and disease modeling purposes. Directed differentiation protocols of iPSC to midbrain dopaminergic neurons enables the study human midbrain Dopamine(DA) neurons, the primary neuronal …
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Modeling Alzheimer's Disease Using CRISPR/CAS9 Gene Editing and Induced Pluripotent Stem Cells Reveals Conserved Cellular Mechanisms
… advances in human induced pluripotent stem cell (iPSC) technology has enabled the study of uniquely human diseases, such as AD, in human tissue. However, the inability to precisely and efficiently genetically engineer human iPSCs has limited their use in effectively studying monogenic human …