Sökning: "rAAV"
Hittade 4 avhandlingar innehållade ordet rAAV.
1. Understanding Cancer Mutations by Genome Editing
Sammanfattning : Mutational analyses of cancer genomes have identified novel candidate cancer genes with hitherto unknown function in cancer. To enable phenotyping of mutations in such genes, we have developed a scalable technology for gene knock-in and knock-out in human somatic cells based on recombination-mediated construct generation and a computational tool to design gene targeting constructs. LÄS MER
2. The Complex Genetics behind Neurodegeneration and Susceptibility to Parkinson’s disease
Sammanfattning : Parkinson’s disease (PD) is the second most common neurodegenerative disorder and affects over 1% of people above the age of 65. This progressive and debilitating disease is usually thought of as a motor disease, with symptoms such as muscle rigidity, slowness of movement, and tremor at rest. LÄS MER
3. Psychiatric and metabolic disturbances in experimental models of Huntington’s disease
Sammanfattning : Non-motor symptoms and signs such as metabolic and psychiatric disturbances have been reported to occur early in Huntington’s disease (HD), a fatal neurodegenerative disorder caused by an expanded CAG repeat in the huntingtin gene. However, there is a lack of understanding of the underlying neurobiological mechanisms responsible for the early non-motor features. LÄS MER
4. 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
