A5001636938- RNA regulation and disease
- Parkinson's Disease Mechanisms and Treatments
- Nerve injury and regeneration
- Neurological disorders and treatments
- Computational Drug Discovery Methods
- Virus-based gene therapy research
- melanin and skin pigmentation
- Antioxidant Activity and Oxidative Stress
- Neuroscience and Neuropharmacology Research
- CRISPR and Genetic Engineering
- Retinoids in leukemia and cellular processes
- 3D Printing in Biomedical Research
- Epilepsy research and treatment
- RNA Interference and Gene Delivery
Research Network (United States)
2023-2024
Biomedical Research Networking Center on Neurodegenerative Diseases
2021-2024
Instituto de Salud Carlos III
2023-2024
Universidad de Navarra
2021-2024
Navarre Institute of Health Research
2021-2022
Although neuromelanin is a dark pigment characteristic of dopaminergic neurons in the human substantia nigra pars compacta, its potential role pathogenesis Parkinson's disease (PD) has often been neglected since most commonly used laboratory animals lack neuromelanin. Here we took advantage adeno-associated viral vectors encoding tyrosinase gene for triggering time-dependent accumulation within compacta macaques up to similar levels pigmentation as observed elderly humans. Furthermore,...
Introduction The presence of a widespread cortical synucleinopathy is the main neuropathological hallmark underlying clinical entities such as Parkinson’s disease with dementia (PDD) and Lewy bodies (DLB). There currently pressing need for development non-human primate (NHPs) models PDD DLB to further overcome existing limitations in drug discovery. Methods Here we took advantage retrogradely-spreading adeno-associated viral vector serotype 9 coding alpha-synuclein A53T mutated gene...
Abstract The SCN1A gene encodes the alpha subunit of a voltage-gated sodium channel (Na v 1.1), which is essential for function inhibitory neurons in brain. Mutations this cause severe encephalopathies such as Dravet syndrome (DS). Upregulation expression by different approaches has demonstrated promising therapeutic effects preclinical models DS. Limiting effect to may contribute restoration brain homeostasis, increasing safety and efficacy treatment. In work, we have evaluated obtain...
Abstract Although neuromelanin (NMel) is a dark pigment characteristic of dopaminergic neurons in the human substantia nigra pars compacta (SNpc), its potential role pathogenesis Parkinson’s disease (PD) has often been neglected since most commonly used laboratory animals lack NMel. Here we took advantage adeno-associated viral vectors encoding tyrosinase gene for triggering time-dependent NMel accumulation within SNpc macaques up to similar levels pigmentation as observed elderly humans....
Abstract The presence of a widespread cortical synucleinopathy is the main neuropathological hallmark underlying clinical entities such as Parkinson’s disease with dementia (PDD) and Lewy bodies (DLB). There currently pressing need for development non-human primate (NHPs) models PDD DLB to further overcome existing limitations in drug discovery. Here we took advantage retrogradely-spreading adeno-associated viral vector serotype 9 coding alpha-synuclein A53T mutated gene induce subcortical...