Sökning: "AAV vectors"
Visar resultat 1 - 5 av 8 avhandlingar innehållade orden AAV vectors.
1. 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
2. Targeted gene transfer in the developing central nervous system using viral vectors
Sammanfattning : Dopamine (DA) is a neurotransmitter that plays a fundamental role in many aspects of normal brain function. The majority of DA neurons in the brain reside in one region ? the midbrain ? and project axonal connections to specific areas. One of the main target regions of the DA system is the striatum. LÄS MER
3. Precision mapping of gene expression and proteins in the brain using gene editing and barcoded viral vectors
Sammanfattning : The human brain is a masterpiece of intricate design and impeccable functionality. It serves as the ultimate command center for our thoughts, sensations, and actions, which define our very existence. LÄS MER
4. Novel strategies for enzyme replacement with gene therapy in models of Parkinson’s disease
Sammanfattning : Parkinson’s disease is a brain disorder characterized by loss of dopaminergic neurons in the midbrain, resulting in the characteristic motor symptoms: resting tremor, rigidity, akinesia and postural instability. Symptomatic treatment is based on reconstituting the loss of dopamine in the brain, primarily by oral administration of its precursor L-DOPA. LÄS MER
5. Restorative and neuroprotective treatment strategies for Parkinson’s disease: tudies on fetal dopamine cell transplants and GDNF delivery by intracerebral injections and recombinant AAV vectors
Sammanfattning : The intrastriatal 6-hydroxydopamine lesion model in rats is particularly useful in studies testing new therapeutic strategies for Parkinson’s disease. This thesis work (1) describes the characterization of the motor deficits after different types of intrastriatal lesions and identifies the four-site terminal lesion as a model of symptomatic Parkinson’s disease; (2) using the same model, provides evidence that the functional impact of the dopamine cell grafts are dependent on interactions with the host brain; (3) demonstrates that protection or restitution of normal motor function after 6-hydroxydopamine lesions can be achieved if glial cell line-derived neurotrophic factor is administered in the striatum at the time of the terminal fiber degeneration. LÄS MER