Sökning: "Avdelningen För Molekylärmedicin Och Genterapi"

Visar resultat 6 - 10 av 48 avhandlingar innehållade orden Avdelningen För Molekylärmedicin Och Genterapi.

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

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

    Sammanfattning : 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. LÄS MER

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

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

    Sammanfattning : 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. LÄS MER

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

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

    Sammanfattning : 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. LÄS MER

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

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

    Sammanfattning : 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. LÄS MER

  7. 10. Hematopoietic stem cell dynamics during regenerative stress

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

    Sammanfattning : .... LÄS MER