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Showing 1 to 20 of 25 for “"induced pluripotent stem cell (iPSC)"”.

  1. The core mammalian pluripotency network in induced pluripotent stem cell (iPSC) formation : models for genetic and epigenetic reprogramming

    … experiment that converted mouse fibroblasts to a pluripotent phenotype coined the 'induced pluripotent stem cell' (iPSC) state. Unhindered by ethical or immunogenic constraints, iPSCs potentially hold the keys to tremendous applications in therapeutic and regenerative medicine. Furthermore, …

    mit Repository record for The core mammalian pluripotency network in induced pluripotent stem cell (iPSC) formation : models for genetic and epigenetic reprogramming (opens in a new tab)

  2. Investigating the role of a FAM111B mutation in hereditary fibrosing poikiloderma (POIKTMP) using induced pluripotent stem cell (iPSC) model

    … the FAM111B mutation (c.1861T>G) in the multi-systemic fibrosis affecting the South African kindred with POIKTMP. Dermal fibroblasts from two affected siblings and a familial control were reprogrammed into induced pluripotent stem cells (iPSCs) via the Sendai virus vector (SeVdp) packaged with …

    cape-town Repository record for Investigating the role of a FAM111B mutation in hereditary fibrosing poikiloderma (POIKTMP) using induced pluripotent stem cell (iPSC) model (opens in a new tab)

  3. Modeling Alzheimer's Disease Using CRISPR/CAS9 Gene Editing and Induced Pluripotent Stem Cells Reveals Conserved Cellular Mechanisms

    … A probable source of this lack of understanding stems from the poor disease model systems that have been utilized in the field for many years. Recently, advances in human induced pluripotent stem cell (iPSC) technology has enabled the study of uniquely human diseases, such as AD, in human tissue. …

    rockefeller Repository record for Modeling Alzheimer's Disease Using CRISPR/CAS9 Gene Editing and Induced Pluripotent Stem Cells Reveals Conserved Cellular Mechanisms (opens in a new tab)

  4. The Functional Role for Chromatin Loops in Gene Expression Control During Human Neuron Maturation

    … in neuroscience, examine neural model systems, and build an auxin-inducible degron to deplete the architectural protein CTCF and disrupt loops genome-wide on short time scales during human induced pluripotent stem cell (iPSC)-derived post-mitotic neuronal maturation. We find thousands of …

    penn Repository record for The Functional Role for Chromatin Loops in Gene Expression Control During Human Neuron Maturation (opens in a new tab)

  5. Development of collagen-based scaffolds for differentiation of induced pluripotent stem cells

    … engineering. A collagen hydrogel can provide cells with a porous and soft environment to proliferate and differentiate. However, lacking mechanical stiffness and shrinkage resistance made it a challenge to sustain shape and size during a long stem cell differentiation process. In addition, a …

    binghamton Repository record for Development of collagen-based scaffolds for differentiation of induced pluripotent stem cells (opens in a new tab)

  6. Systems Biology Approaches for Elucidating Early ALS Disease Processes

    … omics characterization of 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 …

    mit Repository record for Systems Biology Approaches for Elucidating Early ALS Disease Processes (opens in a new tab)

  7. Integrating Omics data : a new software tool and its use in implicating therapeutic targets in Huntington's disease

    … Omics Integrator on omics datasets from induced pluripotent stem cell (iPSC) derived models of HD, I discovered and highlighted several pathways that are altered in these cell line models, including neurodevelopment and glycolytic metabolism, which may lead to important therapeutic …

    mit Repository record for Integrating Omics data : a new software tool and its use in implicating therapeutic targets in Huntington's disease (opens in a new tab)

  8. Novel cell models for the study of spinocerebellar ataxia type 7 pathogenesis and therapy in a South African patient cohort

    … regulation through association with cellular histone acetylation complexes, and several studies have highlighted the role of transcriptional dysregulation, caused by the presence of mutant Ataxin-7, in the neuronal dysfunction that precedes the onset of disease symptoms.This study …

    cape-town Repository record for Novel cell models for the study of spinocerebellar ataxia type 7 pathogenesis and therapy in a South African patient cohort (opens in a new tab)

  9. Engineering Organoids for Stem Cell Maturation

    … and toxicants. Hepatocytes, or the main liver cell type, perform most of these functions. Typically, primary human hepatocytes (PHHs) are ideal for in vitro liver studies since they are obtained directly from tissues and exhibit adult characteristics and functions. However, sourcing these adult …

    vt Repository record for Engineering Organoids for Stem Cell Maturation (opens in a new tab)

  10. From iPSCs to Engineered Heart and Liver Models: Modulating Genetic and Extracellular State for Maturity

    … human physiology yet remain major sites of drug-induced toxicity, driving the need for robust, scalable, and physiologically relevant in vitro models. Induced pluripotent stem cell (iPSC)–derived hepatocyte-like cells (HLCs) and cardiomyocytes (iPSC-CMs) offer patient specificity and renewable …

    uic

  11. Investigating circuit and spatial heterogeneity of human brain tumors with patient-derived organoid models

    … cancers with dismal prognosis. Both malignant cell heterogeneity and the influence of myriad cell types present in the tumor microenvironment, such as neurons, contribute to glioma progression. This dissertation represents a multi-pronged effort to unravel GBM heterogeneity by leveraging …

    penn Repository record for Investigating circuit and spatial heterogeneity of human brain tumors with patient-derived organoid models (opens in a new tab)

  12. MODELING PRO-INFLAMMATORY CROSSTALK BETWEEN MICROGLIA AND ASTROCYTES DURING HIV INFECTION

    … viral transcription in the central nervous system (CNS) and neuroinflammation, though the mechanisms that drive this pathology remain poorly understood. The main cellular host for HIV in the CNS are microglia, which are difficult to model in vitro. We have developed an induced pluripotent stem

    penn Repository record for MODELING PRO-INFLAMMATORY CROSSTALK BETWEEN MICROGLIA AND ASTROCYTES DURING HIV INFECTION (opens in a new tab)

  13. Development of a Cellular Model for Morquio A Syndrome

    … is no applicable animal model or appropriate cell lines currently available for the study of MPS IVA. Furthermore, patient-specific material is limited and difficult to obtain, rendering the study of MPSIVA pathogenesis challenging. Thus, the generation of an induced pluripotent stem cell

    dominican Repository record for Development of a Cellular Model for Morquio A Syndrome (opens in a new tab)

  14. IN VIVO AND IN VITRO EVALUATION OF THE COMBINATION OF RNA INTERFERING AND GENE THERAPY FOR TREATING MITOFUSIN2-RELATED DISEASES

    … essential for mitochondrial networking in most cells. Autosomal dominant mutations in the MFN2 gene cause Charcot-Marie-Tooth type 2A disease (CMT2A), a severe and disabling sensory-motor neuropathy. Here, we propose a novel therapeutic strategy tailored to the correction of the root genetic …

    milano Repository record for IN VIVO AND IN VITRO EVALUATION OF THE COMBINATION OF RNA INTERFERING AND GENE THERAPY FOR TREATING MITOFUSIN2-RELATED DISEASES (opens in a new tab)

  15. Mechanical Property and Phenotype Characterization of an in vitro Model for Afterload-triggered Cardiac Hypertrophy

    … of HCM, in vitro models that use patient-derived induced pluripotent stem cell (iPSC) differentiated cardiomyocytes are especially promising because the unlimited supply of iPSC can allow many studies to be performed under reproducible conditions. In prior studies of afterload-related phenotypes …

    wustl Repository record for Mechanical Property and Phenotype Characterization of an in vitro Model for Afterload-triggered Cardiac Hypertrophy (opens in a new tab)

  16. MODELING AND MODULATING MYELOID CELL-MEDIATED NEUROINFLAMMATION FOLLOWING HUMAN IMMUNODEFICIENCY VIRUS-1 INFECTION

    … immunodeficiency virus 1 (HIV) infects myeloid cells in the central nervous system (CNS) and establishes a reservoir that persists despite antiretroviral therapy (ART). Infection leads to a 30-50% risk of developing HIV-associated neurocognitive disorders (HAND), a collection of mood, motor, and …

    penn Repository record for MODELING AND MODULATING MYELOID CELL-MEDIATED NEUROINFLAMMATION FOLLOWING HUMAN IMMUNODEFICIENCY VIRUS-1 INFECTION (opens in a new tab)

  17. Engineering Three Dimensional In Vitro Approaches to Study Cardiac Injury and Repair

    … infarct, the affected region undergoes massive cell death and a pathological fibrotic response. As cardiomyocytes (CMs) do not regenerate, the CM content is predominately replaced by activated cardiac fibroblasts (CFs). This modifies the regional mechanical and electrical properties, which …

    mit Repository record for Engineering Three Dimensional In Vitro Approaches to Study Cardiac Injury and Repair (opens in a new tab)

  18. Studying lung development in vitro: expanding the genetic toolbox and analysis of SOX2 and SOX9 function

    … is a growing threat to human health worldwide. Induced pluripotent stem cell (iPSC) based stem cell therapy is promising for end-stage respiratory disease patients. Generation of mature/functional lung cells from iPSCs heavily relies on understanding of human lung development. However, analysis …

    cambridge Repository record for Studying lung development in vitro: expanding the genetic toolbox and analysis of SOX2 and SOX9 function (opens in a new tab)

  19. Creation of organoid systems to model human endodermal organ development in vitro

    Organoid systems have revolutionised the study of adult stem cells and organ homeostasis. However, there have been limited attempts to use the technology to study human organ development. The work of this thesis established two novel hepatic organoid systems; one based upon the archetypal liver …

    cambridge Repository record for Creation of organoid systems to model human endodermal organ development in vitro (opens in a new tab)

  20. Modeling Alzheimer's Disease in Induced Pluripotent Stem Cells

    … hallmarks of AD include the deposition of extracellular plaques of aggregated amyloid-β (Aβ) and intracellular neurofibrillary tangles of tau aggregates (NFTs). Autosomal dominant inheritance of AD has been attributed to genetic mutations in three key genes: amyloid precursor protein (APP), …

    rockefeller Repository record for Modeling Alzheimer's Disease in Induced Pluripotent Stem Cells (opens in a new tab)

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