Search Results - "San Sebastian, Waldy"

Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons by Pearson, Toni S., Gupta, Nalin, San Sebastian, Waldy, Imamura-Ching, Jill, Viehoever, Amy, Grijalvo-Perez, Ana, Fay, Alex J., Seth, Neha, Lundy, Shannon M., Seo, Youngho, Pampaloni, Miguel, Hyland, Keith, Smith, Erin, de Oliveira Barbosa, Gardenia, Heathcock, Jill C., Minnema, Amy, Lonser, Russell, Elder, J. Bradley, Leonard, Jeffrey, Larson, Paul, Bankiewicz, Krystof S.

“…Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder characterized by deficient synthesis of dopamine and serotonin. It presents in…”
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AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction by Samaranch, Lluis, Sebastian, Waldy San, Kells, Adrian P, Salegio, Ernesto A, Heller, Gregory, Bringas, John R, Pivirotto, Philip, DeArmond, Stephen, Forsayeth, John, Bankiewicz, Krystof S

“…Many studies have demonstrated that adeno-associated virus serotype 9 (AAV9) transduces astrocytes and neurons when infused into rat or nonhuman primate (NHP)…”
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MR-guided delivery of AAV2-BDNF into the entorhinal cortex of non-human primates by Nagahara, Alan H., Wilson, Bayard R., Ivasyk, Iryna, Kovacs, Imre, Rawalji, Saytam, Bringas, John R., Pivirotto, Phillip J., Sebastian, Waldy San, Samaranch, Lluis, Bankiewicz, Krystof S., Tuszynski, Mark H.

“…Brain-derived neurotrophic factor (BDNF) gene delivery to the entorhinal cortex is a candidate for treatment of Alzheimer’s disease (AD) to reduce…”
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Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS by Naidoo, Jerusha, Stanek, Lisa M., Ohno, Kousaku, Trewman, Savanah, Samaranch, Lluis, Hadaczek, Piotr, O’Riordan, Catherine, Sullivan, Jennifer, San Sebastian, Waldy, Bringas, John R., Snieckus, Christopher, Mahmoodi, Amin, Mahmoodi, Amir, Forsayeth, John, Bankiewicz, Krystof S., Shihabuddin, Lamya S.

“…The present study was designed to characterize transduction of non-human primate brain and spinal cord with a modified adeno-associated virus serotype 2,…”
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Depletion of AADC activity in caudate nucleus and putamen of Parkinson's disease patients; implications for ongoing AAV2-AADC gene therapy trial by Ciesielska, Agnieszka, Samaranch, Lluis, San Sebastian, Waldy, Dickson, Dennis W, Goldman, Samuel, Forsayeth, John, Bankiewicz, Krystof S

“…In Parkinson's disease (PD), aromatic L-amino acid decarboxylase (AADC) is the rate-limiting enzyme in the conversion of L-DOPA (Sinemet) to dopamine (DA)…”
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Distribution of nanoparticles throughout the cerebral cortex of rodents and non-human primates: Implications for gene and drug therapy by Salegio, Ernesto A, Streeter, Hillary, Dube, Nikhil, Hadaczek, Piotr, Samaranch, Lluis, Kells, Adrian P, San Sebastian, Waldy, Zhai, Yuying, Bringas, John, Xu, Ting, Forsayeth, John, Bankiewicz, Krystof S

“…When nanoparticles/proteins are infused into the brain, they are often transported to distal sites in a manner that is dependent both on the characteristics of…”
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Safety and tolerability of MRI-guided infusion of AAV2-hAADC into the mid-brain of nonhuman primate by Sebastian, Waldy San, Kells, Adrian P, Bringas, John, Samaranch, Lluis, Hadaczek, Piotr, Ciesielska, Agnieszka, Macayan, Michael J, Pivirotto, Phillip J, Forsayeth, John, Osborne, Sheryl, Wright, J Fraser, Green, Foad, Heller, Gregory, Bankiewicz, Krystof S

“…Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, autosomal-recessive neurological disorder caused by mutations in the DDC gene that leads to an…”
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Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates by Samaranch, Lluis, Salegio, Ernesto A, San Sebastian, Waldy, Kells, Adrian P, Bringas, John R, Forsayeth, John, Bankiewicz, Krystof S

“…The present study builds on previous work showing that infusion of adeno-associated virus type 9 (AAV9) into the cisterna magna (CM) of nonhuman primates…”
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Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates by Samaranch, Lluis, Salegio, Ernesto A, San Sebastian, Waldy, Kells, Adrian P, Foust, Kevin D, Bringas, John R, Lamarre, Clementine, Forsayeth, John, Kaspar, Brian K, Bankiewicz, Krystof S

“…Widespread distribution of gene products at clinically relevant levels throughout the CNS has been challenging. Adeno-associated virus type 9 (AAV9) vector has…”
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1-Methyl-4-Phenyl-1,2,3,6-Tetrahydropyridine (MPTP) Does Not Elicit Long-Lasting Increases in Cyclooxygenase-2 Expression in Dopaminergic Neurons of Monkeys by Vázquez-Claverie, Marianne, Garrido-Gil, Pablo, San Sebastián, Waldy, Belzunegui, Silvia, Izal-Azcárate, Amaya, López, Berta, Marcilla, Irene, Luquin, María Rosario

“…To elucidate the role of the prostaglandin synthase cyclooxygenase-2 (Cox-2) and the mechanisms of dopaminergic (DA) neurodegeneration, monkeys were injected…”
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Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy by Passini, Marco A, Bu, Jie, Richards, Amy M, Treleaven, Christopher M, Sullivan, Jennifer A, O'Riordan, Catherine R, Scaria, Abraham, Kells, Adrian P, Samaranch, Lluis, San Sebastian, Waldy, Federici, Thais, Fiandaca, Massimo S, Boulis, Nicholas M, Bankiewicz, Krystof S, Shihabuddin, Lamya S, Cheng, Seng H

“…Spinal muscular atrophy (SMA) is a neuromuscular disease caused by mutations in survival motor neuron 1 (SMN1). Previously, we showed that central nervous…”
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An Update on Gene Therapy Approaches for Parkinson's Disease: Restoration of Dopaminergic Function by Van Laar, Amber D, Van Laar, Victor S, San Sebastian, Waldy, Merola, Aristide, Elder, J Bradley, Lonser, Russell R, Bankiewicz, Krystof S

“…At present there is a significant unmet need for clinically available treatments for Parkinson's disease (PD) patients to stably restore balance to dopamine…”
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Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates by Ohno, Kousaku, Samaranch, Lluis, Hadaczek, Piotr, Bringas, John R., Allen, Philip C., Sudhakar, Vivek, Stockinger, Diane E., Snieckus, Christopher, Campagna, Michael V., San Sebastian, Waldy, Naidoo, Jerusha, Chen, Haifeng, Forsayeth, John, Salegio, Ernesto A., Hwa, Granger G.C., Bankiewicz, Krystof S.

“…Here we evaluated the utility of MRI to monitor intrathecal infusions in nonhuman primates. Adeno-associated virus (AAV) spiked with gadoteridol, a…”
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Advances in thin tissue Golgi-Cox impregnation: Fast, reliable methods for multi-assay analyses in rodent and non-human primate brain by Levine, Nathan D., Rademacher, David J., Collier, Timothy J., O’Malley, Jennifer A., Kells, Adrian P., San Sebastian, Waldy, Bankiewicz, Krystof S., Steece-Collier, Kathy

“…► We describe thin tissue ‘Golgi-Cox’ methods that allow fast impregnation without significant crystallization artifact. ► These methods are compatible with…”
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Acute and Chronic 1-Methyl-4-Phenyl-1,2,3,6-Tetrahydropyridine Administrations Elicit Similar Microglial Activation in the Substantia Nigra of Monkeys by Vázquez-Claverie, Marianne, Garrido-Gil, Pablo, San Sebastián, Waldy, Izal-Azcárate, Amaya, Belzunegui, Silvia, Marcilla, Irene, López, Berta, Luquin, María-Rosario

“…Increasing evidence suggests a pivotal role for neuroinflammation in the pathogenesis of Parkinson disease, but whether activated microglia participate in…”
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Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease by Hadaczek, Piotr, Stanek, Lisa, Ciesielska, Agnieszka, Sudhakar, Vivek, Samaranch, Lluis, Pivirotto, Philip, Bringas, John, O'Riordan, Catherine, Mastis, Bryan, San Sebastian, Waldy, Forsayeth, John, Cheng, Seng H, Bankiewicz, Krystof S, Shihabuddin, Lamya S

“…Huntington's disease (HD) is caused by a toxic gain-of-function associated with the expression of the mutant huntingtin (htt) protein. Therefore, the use of…”
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Safety study of adeno-associated virus serotype 2-mediated human acid sphingomyelinase expression in the nonhuman primate brain by Salegio, Ernesto A, Samaranch, Lluis, Jenkins, Russell W, Clarke, Christopher J, Lamarre, Clementine, Beyer, Janine, Kells, Adrian P, Bringas, John, Sebastian, Waldy San, Richardson, R Mark, Rosenbluth, Kate H, Hannun, Yusuf A, Bankiewicz, Krystof S, Forsayeth, John

“…Niemann-Pick disease is a lysosomal storage disorder resulting from inherited deficiency in acid sphingomyelinase (ASM). Use of adeno-associated virus serotype…”
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Immunohistochemical characterization of the rat carotid body by Izal-Azcárate, Amaya, Belzunegui, Silvia, Sebastián, Waldy San, Garrido-Gil, Pablo, Vázquez-Claverie, Marianne, López, Berta, Marcilla, Irene, Luquin, M Rosario

“…Abstract We studied the histochemical phenotype of carotid body (CB) cells in the adult rat. In addition to tyrosine hydroxylase (TH), type I cells expressed…”
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1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine exposure fails to produce delayed degeneration of substantia nigra neurons in monkeys by Garrido-Gil, Pablo, Belzunegui, Silvia, Sebastián, Waldy San, Izal-Azcárate, Amaya, López, Berta, Marcilla, Irene, Luquin, Maria Rosario

“…We assessed the presence of degenerating neurons in the substantia nigra pars compacta (SNpc) and ventral tegmental area (VTA) of parkinsonian monkeys. For…”
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Does increased excitatory drive from the subthalamic nucleus contribute to dopaminergic neuronal death in Parkinson's disease? by Luquin, M. Rosario, Saldise, Laura, Guillén, Javier, Belzunegui, Silvia, San Sebastián, Waldy, Izal, Amaya, Garrido, Pablo, Vázquez, Marianne

“…Excitotoxicity has been suggested to play a pivotal role in the pathogenesis of Parkinson disease (PD). As subthalamic nucleus (STN) neurons express glutamate…”
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