Search Results - "Passini, M A"

Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs by Federici, T, Taub, J S, Baum, G R, Gray, S J, Grieger, J C, Matthews, K A, Handy, C R, Passini, M A, Samulski, R J, Boulis, N M

“…Adeno-associated viral vector 9 (AAV9) has recently been shown to penetrate the blood–brain barrier via intravascular administration, making it a good…”
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Magnetic resonance imaging-guided delivery of adeno-associated virus type 2 to the primate brain for the treatment of lysosomal storage disorders by Salegio, E Aguilar, Kells, A P, Richardson, R M, Hadaczek, P, Forsayeth, J, Bringas, J, Sardi, S P, Passini, M A, Shihabuddin, L S, Cheng, S H, Fiandaca, M S, Bankiewicz, K S

“…Gene replacement therapy for the neurological deficits caused by lysosomal storage disorders, such as in Niemann-Pick disease type A, will require widespread…”
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Long-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters by Husain, T, Passini, M A, Parente, M K, Fraser, N W, Wolfe, J H

“…The neurogenetic, lysosomal enzyme (LSE) deficiency diseases are characterized by storage lesions throughout the brain; therefore, gene transfer needs to…”
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Intracerebral Transplantation of Adult Mouse Neural Progenitor Cells into the Niemann-Pick-A Mouse Leads to a Marked Decrease in Lysosomal Storage Pathology by Shihabuddin, L. S, Numan, S, Huff, M. R, Dodge, J. C, Clarke, J, Macauley, S. L, Yang, W, Taksir, T. V, Parsons, G, Passini, M. A, Gage, F. H, Stewart, G. R

“…Niemann-Pick disease is caused by a genetic deficiency in acid sphingomyelinase (ASM) leading to the intracellular accumulation of sphingomyelin and…”
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Widespread Gene Delivery and Structure-Specific Patterns of Expression in the Brain after Intraventricular Injections of Neonatal Mice with an Adeno-Associated Virus Vector by Passini, M A, Wolfe, J H

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Cloning of Zebrafish Neurofilament cDNAs for Plasticin and Gefiltin: Increased mRNA Expression in Ganglion Cells After Optic Nerve Injury by Asch, William S., Leake, Devin, Canger, Anthony K., Passini, Marco A., Argenton, Francesco, Schechter, Nisson

“…: During retinal growth and optic axon regeneration, the differential expression of the neuronal intermediate filament proteins, plasticin and gefiltin, in the…”
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Vsx-1 and Vsx-2: Differential expression of two Paired-like homeobox genes during zebrafish and goldfish retinogenesis by Passini, Marco A., Levine, Edward M., Canger, Anthony K., Raymond, Pamela A., Schechter, Nisson

“…Vsx‐1 and Vsx‐2 are two homeobox genes that were cloned originally from an adult goldfish retinal library. They are members of the paired‐like:CVC gene family,…”
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Vsx-2, a gene encoding a paired-type homeodomain, is expressed in the retina, hindbrain, and spinal cord during goldfish embryogenesis by Passini, Marco A, Raymond, Pamela A, Schechter, Nisson

“…Vsx-2 encodes a paired-type homeodomain and is the goldfish ortholog of the murine Chx10 gene. During development, Vsx-2 is expressed at high levels in…”
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Restricted expression of the neuronal intermediate filament protein plasticin during zebrafish development by Canger, Anthony K., Passini, Marco, Asch, William S., Leake, Devin, Zafonte, Brian T., Glasgow, Eric, Schechter, Nisson

“…In the adult goldfish visual pathway, expression of the neuronal intermediate filament (nIF) protein plasticin is restricted to differentiating retinal…”
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CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy by Passini, Marco A, Bu, Jie, Roskelley, Eric M, Richards, Amy M, Sardi, S Pablo, O'Riordan, Catherine R, Klinger, Katherine W, Shihabuddin, Lamya S, Cheng, Seng H

“…Spinal muscular atrophy (SMA) is a neuromuscular disease caused by a deficiency of survival motor neuron (SMN) due to mutations in the SMN1 gene. In this…”
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Long-term AA V vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters by HUSAIN, T, PASSINI, M. A, PARENTE, M. K, FRASER, N. W, WOLFE, J. H

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Intraventricular Brain Injection of Adeno-Associated Virus Type 1 (AAV1) in Neonatal Mice Results in Complementary Patterns of Neuronal Transduction to AAV2 and Total Long-Term Correction of Storage Lesions in the Brains of β-Glucuronidase-Deficient Mice by Passini, Marco A, Watson, Deborah J, Vite, Charles H, Landsburg, Daniel J, Feigenbaum, Alyson L, Wolfe, John H

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Intracranial Delivery of CLN2 Reduces Brain Pathology in a Mouse Model of Classical Late Infantile Neuronal Ceroid Lipofuscinosis by Passini, Marco A, Dodge, James C, Bu, Jie, Yang, Wendy, Zhao, Qi, Sondhi, Dolan, Hackett, Neil R, Kaminsky, Stephen M, Mao, Qinwen, Shihabuddin, Lamya S, Cheng, Seng H, Sleat, David E, Stewart, Gregory R, Davidson, Beverly L, Lobel, Peter, Crystal, Ronald G

“…Classical late infantile neuronal ceroid lipofuscinosis (cLINCL) is a lysosomal storage disorder caused by mutations in CLN2, which encodes lysosomal…”
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Effective gene therapy for an inherited CNS disease in a large animal model by Vite, Charles H., McGowan, Joseph C., Niogi, Sumit N., Passini, Marco A., Drobatz, Kenneth J., Haskins, Mark E., Wolfe, John H.

“…Genetic diseases affecting the brain typically have widespread lesions that require global correction. Lysosomal storage diseases are good candidates for…”
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Targeted Transduction Patterns in the Mouse Brain by Lentivirus Vectors Pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV Envelope Proteins by Watson, Deborah J., Kobinger, Gary P., Passini, Marco A., Wilson, James M., Wolfe, John H.

“…Lentiviral vectors have proven to be promising tools for transduction of central nervous system (CNS) cells in vivo and in vitro. In this study, CNS…”
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A Mouse Model of Classical Late-Infantile Neuronal Ceroid Lipofuscinosis Based on Targeted Disruption of the CLN2 Gene Results in a Loss of Tripeptidyl-Peptidase I Activity and Progressive Neurodegeneration by Sleat, David E, Wiseman, Jennifer A, El-Banna, Mukarram, Kim, Kwi-Hye, Mao, Qinwen, Price, Sandy, Macauley, Shannon L, Sidman, Richard L, Shen, Michael M, Zhao, Qi, Passini, Marco A, Davidson, Beverly L, Stewart, Gregory R, Lobel, Peter

“…Mutations in the CLN2 gene, which encodes a lysosomal serine protease, tripeptidyl-peptidase I (TPP I), result in an autosomal recessive neurodegenerative…”
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Egr3 stimulation of GABRA4 promoter activity as a mechanism for seizure-induced up-regulation of GABA(A) receptor alpha4 subunit expression by Roberts, D S, Raol, Y H, Bandyopadhyay, S, Lund, I V, Budreck, E C, Passini, M A, Passini, M J, Wolfe, J H, Brooks-Kayal, A R, Russek, S J

“…GABA is the major inhibitory transmitter at CNS synapses. Changes in subunit composition of the pentameric GABA(A) receptor, including increased levels of…”
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AAV-Mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy by Rafi, Mohammad A., Zhi Rao, Han, Passini, Marco A., Curtis, Mark, Vanier, Marie T., Zaka, Mariam, Luzi, Paola, Wolfe, John H., Wenger, David A.

“…Globoid cell leukodystrophy (GLD) or Krabbe disease is a neurodegenerative disorder caused by a deficiency of galactocerebrosidase (GALC) activity. GALC is…”
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Distribution of a Lysosomal Enzyme in the Adult Brain by Axonal Transport and by Cells of the Rostral Migratory Stream by Passini, Marco A, Lee, Edward B, Heuer, Gregory G, Wolfe, John H

“…A portion of the lysosomal enzymes produced by cells is secreted, diffuses through extracellular spaces, and can be taken up by distal cells via…”
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Adeno-associated virus vector-mediated transduction in the cat brain by VITE, Charles H, PASSINI, Marco A, HASKINS, Mark E, WOLFE, John H

“…Adeno-associated virus (AAV) vectors are capable of delivering a therapeutic gene to the mouse brain that can result in long-term and widespread protein…”
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