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

Showing result 6 - 10 of 48 swedish dissertations containing the words Avdelningen för molekylärmedicin och genterapi.

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

    Author : Alexandra Bäckström; Avdelningen för molekylärmedicin och genterapi; []
    Keywords : 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

  4. 7. 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; 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

  5. 8. Modelling Human Developmental Hematopoiesis. Towards in vitro Generation of Human Hematopoietic Stem Cells

    Author : Carolina Guibentif; Avdelningen för molekylärmedicin och genterapi; []
    Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; NATURVETENSKAP; NATURAL SCIENCES; Human pluripotent stem cells; Embryonic Development; endothelial-to-hematopoietic transition; Hematopoiesis;

    Abstract : The possibility to manufacture hematopoietic stem cells (HSCs) in the laboratory would provide an indefinite source of cells for patients requiring bone marrow transplantation. Moreover, combined with the progress in gene editing techniques, it would provide a novel platform for gene and cell replacement therapies for a range of currently incurable congenic and acquired disorders. READ MORE

  6. 9. Development of Novel Therapies, Models, and Biomarkers for Osteoclast-Related Diseases

    Author : Henrik Löfvall; Avdelningen för molekylärmedicin och genterapi; []
    Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Osteopetros; Biomarkör; Cellodling; Extracellulär matrix ECM ; Osteoklast; Osteoclast; Biomarker; Cell culture; Osteopetrosis; Gene Therapy; Extracellular matrix ECM ;

    Abstract : Osteoclasts are derived from hematopoietic stem cells (HSCs) and form upon stimulation of osteoclast precursors by macrophage colony-stimulating factor (M-CSF) and receptor activator of nuclear factor κΒ ligand (RANKL). Osteoclasts resorb bone by secreting hydrochloric acid, matrix metalloproteinases (MMPs) and cathepsin K. READ MORE

  7. 10. Hematopoietic stem cell dynamics during regenerative stress

    Author : Anna Rydström; Avdelningen för molekylärmedicin och genterapi; []
    Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; hematopoietic stem cell; transplantation; regeneration; stress;

    Abstract : .... READ MORE