Search for dissertations about: "AAV vectors"

Showing result 1 - 5 of 8 swedish dissertations containing the words AAV vectors.

2. 1. 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

4. 2. Targeted gene transfer in the developing central nervous system using viral vectors

   Author : Simon Stott; Brain Repair and Imaging in Neural Systems (BRAINS); []
   Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Neurology; neuropsychology; neurophysiology; Neurologi; Medicin människa och djur ; Medicine human and vertebrates ; gene transfer; in utero; neonatal; GDNF; nigrostriatal; dopamine; AAV; retrovirus; neuropsykologi; neurofysiologi;

   Abstract : 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. READ MORE

5. 3. Precision mapping of gene expression and proteins in the brain using gene editing and barcoded viral vectors

   Author : Martino Avallone; Molekylär neuromodulering; []
   Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; Gene editing; CRISPR Cas9; Barcodes; AAV; PLA; gene therapy; Parkinson’s disease; single nuclei RNAseq; alpha-synuclein;

   Abstract : 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. READ MORE

6. 4. Novel strategies for enzyme replacement with gene therapy in models of Parkinson’s disease

   Author : Erik Cederfjäll; Brain Repair and Imaging in Neural Systems (BRAINS); []
   Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; AAV vectors; dopamine; enzyme replacement; gene regulation; GTP cyclohydrolase 1; L-DOPA; tyrosine hydroxylase; Parkinson’s disease;

   Abstract : 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. READ MORE

7. 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

   Author : Deniz Kirik; Neurobiologi; []
   Keywords : MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; adeno-associated virus; regeneration; neuroprotection; glial-cell-line-derived-neurotrophic factor; cell death; substantia nigra; dopamine; neurofysiologi; neuropsykologi; Neurologi; neurophysiology; neuropsychology; Neurology; tyrosine hydroxylase; sensorimotor behavior; transplantation; gene therapy;

   Abstract : 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. READ MORE