Search for dissertations about: "Lentiviral Vectors"

Showing result 1 - 5 of 19 swedish dissertations containing the words Lentiviral Vectors.

2. 1. 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; 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

4. 2. Generation of induced neurons via direct conversion in vivo and in vitro

   Author : Olof Torper; Neurobiologi; []
   Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Cellular reprogramming; cell replacement therapies; induced dopaminergic neurons; in vivo reprogramming; Lentiviral vectors; AAV vectors;

   Abstract : Cellular reprogramming is when one cell is changed into another. This involves structural modifications on the DNA of a cell resulting in a transcriptional change. This occurs naturally during development when early pluripotent cells gradually differentiate into more specialized cells that finally result in a complete organism. READ MORE

5. 3. Development of lentiviral vectors for CNS gene transfer

   Author : Johan Jakobsson; Brain Repair and Imaging in Neural Systems (BRAINS); []
   Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Medicin människa och djur ; Medicine human and vertebrates ; Biotechnology; Gene Therapy; Neurological disorders;

   Abstract : Gene therapy in the brain is a promising treatment strategy that in the future may be used for several brain disorders, including Parkinson's disease, Alzheimer's disease and lysosomal storage diseases. By introducing a new gene, rather than providing a classical pharmacological drug, gene therapy offers the opportunity to treat neurological disorders by a single intervention. READ MORE

6. 4. 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

7. 5. Alphavirus vectors as recombinant vaccines

   Author : Peter Berglund; Karolinska Institutet; Karolinska Institutet; []
   Keywords : Semliki Forest virus; Alphavirus; Synthetic vaccmes; Influenza A virus; HIV-I; SIV; DNA vaccines; AIDS vaccines; Influenza vaccine;

   Abstract : This thesis describes further developments of an expression system based on the alphavirus Semliki Forest virus (SFV), and its potential use as recombinant vaccine. The RNA genome of SFV contains a 3'open reading frame (ORF) encoding the structural proteins, and a 5' ORF coding for the viral replicase. READ MORE