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 20 of 157 for “"iPSC"”.
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Systematic Co-Culture of IPSC-CM and IPSC-AN Promote Co-Maturation In Vitro
… autonomic neuron differentiation efficiency (iPSC-ANs), establish a platform for CM/AN co-culture, and investigate the influence of these parameters on CM/AN crosstalk/responses.</p> <p>A protocol was developed in-house that incorporated <em>in vivo</em> developmental cues. The differentiated …
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Monte Carlo simulation of radiation transport for benchmarking Intel's iPSC/2, IPSC/860, and Touchstone Delta machine
Thesis (M. Eng.)--Massachusetts Institute of Technology, Dept. of Electrical Engineering and Computer Science, 1994.
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Modelling neurodegenerative diseases in human iPSC-derived neurons
… of in vitro disease models using human iPSC-derived neurons to provide a more accurate understanding of how disease mutations affect neuronal health. I first sought to model the polyglutamine disease spinal and bulbar muscular atrophy (SBMA), detailed in Chapter 4. Using iPSCs derived …
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DISSECTING THE MOLECULAR MECHANISMS RELATED TO THE ONSET OF NEUROLOGICAL COMPLICATIONS IN THREE CELLULAR MODELS OF SARS-COV 2 INFECTION: IPSC-DERIVED MOTO R NEURONS, IPSC-DERIVED DOPAMINERGIC NEURONS, IPSC DERIVED-HUMAN CORTICAL ORGANOIDS.
… neuronal models, including dopaminergic neurons, iPSC derived motor neurons, and human cortical organoids, in order to investigate this extremely current and concerning condition. Each of these projects, addressed below in the text, has independently contributed to broadening knowledge about the …
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Modeling colorectal cancer initiation using human iPSC-derived intestinal organoids
OBJECTIVE: Human induced pluripotent stem cell (hiPSC)-derived human intestinal organoids (HIOs) are increasingly utilized as in-vitro models for studying colorectal cancer (CRC). Prevalent oncogenic drivers in CRC include mutations in EGFR (L858R), KRAS (G12D) and CTNNB1 (S33C) which are known to …
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Phenotypic characterisation of human iPSC neuronal models of GM2 gangliosidoses
Gangliosides are crucial components on the outer leaflet of the plasma membrane of many cells, especially neurons. Their functions are broad and varied but their high abundance in neurons leaves these cells especially vulnerable to the effects of their accumulation in ganglioside lysosomal storage …
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EPIGENETIC REGULATION IN AN IPSC-NEURON MODEL OF FAMILIAL ALZHEIMER’S DISEASE
… non-demented controls. In this thesis, I use iPSC-neurons derived from familial AD patients with an APP duplication as a model to study the functional effect of H3K27ac reduction. I found that in iPSC-neurons derived from the AD patients, homeostatic amyloid-reducing genes were upregulated …
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Cellular Phenotypes Associated with NRXN1 Mutations in Autism; an iPSC Study
… derived from induced pluripotent stem cells (iPSC) using the OPTi-OX system to study the impact of NRXN1 mutations on excitatory glutamatergic neurons. I specifically study the molecular, morphological and functional changes caused by 1) induced mutations in CRISPR-edited cell lines; and 2) …
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Master regulators of neuronal maturation in NGN2 reprogrammed iPSC-derived iNeurons
Neuronal maturation is an intricate and tightly-controlled process in neurodevelopment, during which neuronal cells acquire their mature molecular, morphological and electrophysiological properties to function as the central components of the nervous system. However, the cell-intrinsic mechanisms …
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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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Development of Novel Therapies for Marfan Syndrome using a Human iPSC-disease model
… in the pathogenicity. Here, I use a patient iPSC-derived in vitro disease model that recapitulates the complexity of the patient abnormalities (Granata et al. 2017) to develop two potential therapeutic strategies (i) directly interfering at the level of the pathological mutation using an exon …
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MODELLING RIBOFLAVIN TRANSPORTER DEFICIENCY (RTD) USING IPSC-DERIVED MODELS TO TEST GENE THERAPY EFFICACY
… of RTD patients-specific induced pluripotent (iPSC) line carrying several mutations in SLC52A2 gene. First, we tested gene replacement therapy on RTD type 2 patient-derived motoneurons using an adeno-associated viral vector 2/9 (AAV9) carrying the human codon optimized SLC52A2 cDNA. We …
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INDUCED PLURIPOTENT STEM CELLS (IPSC) TO STUDY PATHOMECHANISMS ASSOCIATED TO AMYOTROPHIC LATERAL SCLEROSIS (ALS)
… patient-derived induced pluripotent stem cells (iPSC) represents a great advancement to study neurodegenerative disorders, because iPSC maintain the individual genetic background and can be differentiated into different neuro- glial cell lineages, including motoneurons and cortical neurons. In my …
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Increased Oxidative Phosphorylation In An Ipsc- Derived Model of Rothmund-Thomson Syndrome Associated Osteosarcomagenesis
… of 12 induced pluripotent stem cell lines (iPSCs) from two paired RTS patient/ parental control (Family) sets. These iPSCs were validated, then differentiated to the bone precursor, mesenchymal stem cells (MSCs). After verification of the MSCs, we differentiated the MSCs to osteoblasts, …
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Development of a High-Throughput Human iPSC Chondrogenesis Platform and Applications for Arthritis Disease Modeling
… of human induced pluripotent stem cells (hiPSCs) to prescribed cell fates enables the engineering of patient-specific tissue types, such as hyaline cartilage, for applications in regenerative medicine, disease modeling, and drug screening. In many cases, however, these differentiation …
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Creating CRISPR-Cas9 genome edited iPSC lines to model a patient-specific mutation in mitochondrial disease
… editing in induced pluripotent stem cells (iPSC) provides an opportunity to model mt-aaRS mutations in vitro and investigate the effects of individual mutations on cellular phenotype. SARS2 encodes mitochondrial seryl tRNA-synthetase, and its c.1347 G>A mutation causes severe childhood-onset …
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Lineage-Specific Phenotypic Abnormalities in iPSC-Derived Smooth Muscle Cells from Type-I Loeys-Dietz Syndrome
… patient-derived induced pluripotent stem cells (iPSCs) carrying a heterozygous TGFBR1+/R487Q mutation and its mutation-corrected isogenic control iPSCs to model Type-I LDS. Phenotypic characterisation, such as by bulk RNA sequencing, revealed that LM-SMCs have abnormal phenotypes, including …
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Parkinson's Disease-Associated Protein VPS13C Regulates Phospho-Rab10-Mediated Lysosomal Function in Human iPSC-Derived Dopaminergic Neurons
Neurodegenerative diseases are progressive disorders with age as the greatest risk factor. Parkinson’s disease (PD) is the most common movement disorder and the second most common neurodegenerative disease after Alzheimer’s disease. A stark increase in the incidence of neurodegenerative diseases is …
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Ipsc Based Gene Correction and Disease Model of A New Class of Lgmd Due to Poglut1 Mutation
… progenitors.</p> <p>To test this, we have used iPSCs for disease modeling and rescue experiments. Using a CRISPR based gene targeting method, we aimed to correct the point mutation and restore POGLUT1 function, thus restoring Notch signaling activity. Following correction, iPSC-derived gene …
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