Search for dissertations about: "Avdelningen för molekylärmedicin och genterapi"

Showing result 1 - 5 of 45 swedish dissertations containing the words Avdelningen för molekylärmedicin och genterapi.

  2. 1. The Effects of Genetic and Epigenetic Variation on Human Pluripotent Stem Cell Differentiation

    Author : Roksana Moraghebi; Avdelningen för molekylärmedicin och genterapi; []
    Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES;

    Abstract : Human pluripotent stem cells (PSCs) are widely used for studying embryonic development, disease modelling, drug discovery and cell therapy development. Using human PSCs as a model has significantly contributed to our understanding of embryonic development and elucidating novel pathological mechanisms as well as developing new drugs. READ MORE

  4. 2. Generating Dendritic Cells by Direct Cell Reprogramming. Merging cellular reprogramming with immunology towards the development of novel cancer immunotherapies

    Author : Fábio Fiúza Rosa; Avdelningen för molekylärmedicin och genterapi; []
    Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Dendritic Cell; Direct Cell Reprogramming; Single-cell analysis; Immunotherapy;

    Abstract : Cell fate reprogramming towards pluripotency or alternative somatic cell-types has highlighted the plasticity of adult somatic cells, providing new technologies to generate desired cell types for tissue repair or for disease modeling. There is momentum to bring these concepts to immunology by specifying unique immune cellular identities that set in motion immune responses. READ MORE

  5. 3. Development of Gene Therapy for Hematopoietic Stem Cells using Lentiviral Vectors

    Author : Niels-Bjarne Woods; Andreas Ooka; Stefan Karlsson; Avdelningen för molekylärmedicin och genterapi; []
    Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; embryonic stem cell; Hematopoietic stem cell; lentiviral vector; transduction efficiency; transgene expression; multiple vector copy integration; insertional mutagenesis; NOD SCID mouse; cytogenetics; Genetics; extracellulära vätskor; extracellular fluids; cytogenetik; Genetik; Haematology; Hematologi; Lentivirus : genetics; Human; Genetic Vectors; Hematopoietic Stem Cells : metabolism; Animal; Gene Therapy; Leukemia : therapy; Leukemia : metabolism;

    Abstract : Hematopoietic stem cells are an ideal target for genetic manipulation for the purpose of curing hematological disorders as they have the ability to develop into all blood lineages and to self-renew. In this study we demonstrate that lentiviral vectors, based on HIV-1, can efficiently transfer genes into human hematopoietic progenitor and stem cells as assessed following stem cell transplantation in immune compromised mice. READ MORE

  6. 4. Modeling Human Hematopoiesis Using the CRISPR/Cas9 System

    Author : Alexandra Bäckström; Avdelningen för molekylärmedicin och genterapi; []
    Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Hematopoiesis; Hematopoietic stem and progenitor cells; HSC regulation; CRISPR Cas9; Gene editing;

    Abstract : Hematopoietic stem cells (HSCs) have the ability to self-renew and to give rise to all blood cells of the different lineages, and are thereby responsible for the replenishment of blood cells throughout life. These cells are tightly regulated by extrinsic and intrinsic regulators, such as signals from the bone marrow microenvironment, complex transcription factor networks and epigenetic regulators. READ MORE

  7. 5. Cell and gene therapy for blood disorders. Modeling of novel treatment strategies for Gaucher type 1 disease and Diamond-Blackfan anemia in mice

    Author : Maria Dahl; Avdelningen för molekylärmedicin och genterapi; []
    Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Gaucher disease; lentiviral vectors; gene therapy; Diamond-Blackfan anemia; hematopoietic stem cells; bone marrow transplant; Schlafen-2;

    Abstract : Gaucher disease type 1 (GD type 1) and Diamond-Blackfan anemia (DBA) are rare genetic disorders affecting the hematopoietic system and routinely treated by administration of drugs with variable clinical efficacy. Though effective in alleviating disease symptoms in large cohorts of patients, these treatment options have the major drawback of being non-curative. READ MORE