Sökning: "Lentiviral Vectors"
Visar resultat 1 - 5 av 19 avhandlingar innehållade orden Lentiviral Vectors.
1. Development of Gene Therapy for Hematopoietic Stem Cells using Lentiviral Vectors
Sammanfattning : 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. LÄS MER
2. Generation of induced neurons via direct conversion in vivo and in vitro
Sammanfattning : 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. LÄS MER
3. Development of lentiviral vectors for CNS gene transfer
Sammanfattning : 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. LÄS MER
4. Cell and gene therapy for blood disorders. Modeling of novel treatment strategies for Gaucher type 1 disease and Diamond-Blackfan anemia in mice
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. Alphavirus vectors as recombinant vaccines
Sammanfattning : 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. LÄS MER