Sökning: "Molekylärmedicin"

Visar resultat 36 - 40 av 74 avhandlingar innehållade ordet Molekylärmedicin.

1. 36. 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. 37. Programming Blood Cell Fates. Insights from Direct Lineage Conversion and Development

   Författare :Sandra Capellera Garcia; Avdelningen för molekylärmedicin och genterapi; []
   Nyckelord :MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Hematopoietic development; Erythropoiesis; Megakaryopoiesis; Direct Lineage Conversion;

   Sammanfattning : Red blood cells (RBC) and platelets constitute the non-immune branch of the hematopoietic system and are responsible for the vital functions of transporting oxygen to the tissues and clotting blood vessel injuries, respectively. These cells are produced during embryonic development and throughout life through a process called hematopoiesis which is tightly regulated by extrinsic and intrinsic factors. LÄS MER

4. 38. 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

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

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

   Sammanfattning : 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. LÄS MER

7. 40. Targeting the hematopoietic stem cell to correct osteopetrosis

   Författare :Carmen Flores Bjurström; Avdelningen för molekylärmedicin och genterapi; []
   Nyckelord :MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Hematopoietic stem cells; bone marrow transplantation; gene therapy; osteoclasts; infantile malignant osteopetrosis;

   Sammanfattning : This thesis focuses on developing stem cell targeted gene therapy for the severe hereditary disorder Infantile Malignant Osteopetrosis (IMO) as well as increasing the understanding of how the genetic defect present in IMO affects the hematopoietic stem cells and hematopoiesis in general. IMO is a rare congenital disorder associated with an increased number of non-functional osteoclasts and the absence of bone resorption results in accumulation of sclerotic bone leading to abnormal bone marrow cavity formation insufficient to support hematopoiesis. LÄS MER