Sökning: "lentiviral vector"
Visar resultat 1 - 5 av 18 avhandlingar innehållade orden lentiviral vector.
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. GDNF gene delivery in an animal model of Parkinson's disease. Long-term effects on intact, injured and transplanted dopamine neurons using lentiviral gene transfer
Sammanfattning : Parkinson's disease is characterized by a progressive degeneration of dopaminergic neurons in the substantia nigra, leading to a loss of dopamine in the target structure striatum and development of motor symptoms, such as bradykinesia, rigidity and tremor. New experimental treatment strategies for Parkinson's disease are aimed at either preventing the degeneration of the dopaminergic neurons, or at restoring dopamine in the striatum by fetal dopaminergic transplants. LÄS MER
4. Modifying xenogeneic immune recognition and engraftment by genetic engineering
Sammanfattning : Transplantation using xenogenic organs, tissues and cells (i.e. xenotransplantation) is a potential solution to the shortage of those from human sources. Vascular endothelial cells (ECs) are the most immediate barrier between the xenogeneic donor organ and the host defense systems. LÄS MER
5. Specific genetic modifications in the CNS - Cell specific expression and gene specific regulation
Sammanfattning : Gene therapy is a promising therapeutic tool for many diseases of the CNS. Lentiviral vectors are particularly attractive since this vector type can transduce both dividing and non-dividing cells, have a relatively large cloning capacity, can sustain long-term transgene expression and have low immunogenicity. LÄS MER