Search Results - "Vite, Charles H"

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals by Hinderer, Christian, Bell, Peter, Katz, Nathan, Vite, Charles H, Louboutin, Jean-Pierre, Bote, Erin, Yu, Hongwei, Zhu, Yanqing, Casal, Margret L, Bagel, Jessica, O'Donnell, Patricia, Wang, Ping, Haskins, Mark E, Goode, Tamara, Wilson, James M

“…Delivery of adeno-associated viral (AAV) vectors into the cerebrospinal fluid (CSF) can achieve gene transfer to cells throughout the brain and spinal cord,…”
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Krabbe disease successfully treated via monotherapy of intrathecal gene therapy by Bradbury, Allison M, Bagel, Jessica H, Nguyen, Duc, Lykken, Erik A, Pesayco Salvador, Jill, Jiang, Xuntian, Swain, Gary P, Assenmacher, Charles A, Hendricks, Ian J, Miyadera, Keiko, Hess, Rebecka S, Ostrager, Arielle, ODonnell, Patricia, Sands, Mark S, Ory, Daniel S, Shelton, G Diane, Bongarzone, Ernesto R, Gray, Steven J, Vite, Charles H

“…Globoid cell leukodystrophy (GLD; Krabbe disease) is a progressive, incurable neurodegenerative disease caused by deficient activity of the hydrolytic enzyme…”
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Efficacy and Safety of a Krabbe Disease Gene Therapy by Hordeaux, Juliette, Jeffrey, Brianne A, Jian, Jinlong, Choudhury, Gourav R, Michalson, Kristofer, Mitchell, Thomas W, Buza, Elizabeth L, Chichester, Jessica, Dyer, Cecilia, Bagel, Jessica, Vite, Charles H, Bradbury, Allison M, Wilson, James M

“…Krabbe disease is a lysosomal storage disease caused by mutations in the gene that encodes galactosylceramidase, in which galactosylsphingosine (psychosine)…”
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Accumulation of alkyl-lysophosphatidylcholines in Niemann-Pick disease type C1 by Mishra, Sonali, Kell, Pamela, Scherrer, David, Dietzen, Dennis J., Vite, Charles H., Berry-Kravis, Elizabeth, Davidson, Cristin, Cologna, Stephanie M., Porter, Forbes D., Ory, Daniel S., Jiang, Xuntian

“…Lysosomal function is impaired in Niemann-Pick disease type C1 (NPC1), a rare and inherited neurodegenerative disorder, resulting in late endosomal/lysosomal…”
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Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease by Vite, Charles H, Bagel, Jessica H, Swain, Gary P, Prociuk, Maria, Sikora, Tracey U, Stein, Veronika M, O'Donnell, Patricia, Ruane, Therese, Ward, Sarah, Crooks, Alexandra, Li, Su, Mauldin, Elizabeth, Stellar, Susan, De Meulder, Marc, Kao, Mark L, Ory, Daniel S, Davidson, Cristin, Vanier, Marie T, Walkley, Steven U

“…Niemann-Pick type C1 (NPC) disease is a lysosomal storage disease caused by mutations in the NPC1 gene, leading to an increase in unesterified cholesterol and…”
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Forecasting seizures in dogs with naturally occurring epilepsy by Howbert, J Jeffry, Patterson, Edward E, Stead, S Matt, Brinkmann, Ben, Vasoli, Vincent, Crepeau, Daniel, Vite, Charles H, Sturges, Beverly, Ruedebusch, Vanessa, Mavoori, Jaideep, Leyde, Kent, Sheffield, W Douglas, Litt, Brian, Worrell, Gregory A

“…Seizure forecasting has the potential to create new therapeutic strategies for epilepsy, such as providing patient warnings and delivering preemptive therapy…”
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AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease) by Bradbury, Allison M, Rafi, Mohammed A, Bagel, Jessica H, Brisson, Becky K, Marshall, Michael S, Pesayco Salvador, Jill, Jiang, Xuntain, Swain, Gary P, Prociuk, Maria L, ODonnell, Patricia A, Fitzgerald, Caitlin, Ory, Daniel S, Bongarzone, Ernesto R, Shelton, G Diane, Wenger, David A, Vite, Charles H

“…Globoid cell leukodystrophy (GLD), or Krabbe disease, is an inherited, neurologic disorder that results from deficiency of a lysosomal enzyme,…”
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N-acyl-O-phosphocholineserines: structures of a novel class of lipids that are biomarkers for Niemann-Pick C1 disease by Sidhu, Rohini, Mondjinou, Yawo, Qian, Mingxing, Song, Haowei, Kumar, Arun Babu, Hong, Xinying, Hsu, Fong-Fu, Dietzen, Dennis J., Yanjanin, Nicole M., Porter, Forbes D., Berry-Kravis, Elizabeth, Vite, Charles H., Gelb, Michael H., Schaffer, Jean E., Ory, Daniel S., Jiang, Xuntian

“…Niemann-Pick disease type C1 (NPC1) is a fatal, neurodegenerative, cholesterol storage disorder. With new therapeutics in clinical trials, there is an urgency…”
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Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy by Yoon, Sea Young, Hunter, Jacqueline E, Chawla, Sanjeev, Clarke, Dana L, Molony, Caitlyn, O'Donnell, Patricia A, Bagel, Jessica H, Kumar, Manoj, Poptani, Harish, Vite, Charles H, Wolfe, John H

“…Intravascular injection of certain adeno-associated virus vector serotypes can cross the blood-brain barrier to deliver a gene into the CNS. However, gene…”
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Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII by Gurda, Brittney L, De Guilhem De Lataillade, Adrien, Bell, Peter, Zhu, Yanqing, Yu, Hongwei, Wang, Ping, Bagel, Jessica, Vite, Charles H, Sikora, Tracey, Hinderer, Christian, Calcedo, Roberto, Yox, Alexander D, Steet, Richard A, Ruane, Therese, O'Donnell, Patricia, Gao, Guangping, Wilson, James M, Casal, Margret, Ponder, Katherine P, Haskins, Mark E

“…Mucopolysaccharidosis VII (MPS VII) is a lysosomal storage disease arising from mutations in β-d-glucuronidase (GUSB), which results in glycosaminoglycan (GAG)…”
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A digital atlas of the dog brain by Datta, Ritobrato, Lee, Jongho, Duda, Jeffrey, Avants, Brian B, Vite, Charles H, Tseng, Ben, Gee, James C, Aguirre, Gustavo D, Aguirre, Geoffrey K

“…There is a long history and a growing interest in the canine as a subject of study in neuroscience research and in translational neurology. In the last few…”
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Application of N-palmitoyl-O-phosphocholineserine for diagnosis and assessment of response to treatment in Niemann-Pick type C disease by Sidhu, Rohini, Kell, Pamela, Dietzen, Dennis J., Farhat, Nicole Y., Do, An Ngoc Dang, Porter, Forbes D., Berry-Kravis, Elizabeth, Vite, Charles H., Reunert, Janine, Marquardt, Thorsten, Giugliani, Roberto, Lourenço, Charles M., Bodamer, Olaf, Wang, Raymond Y., Plummer, Ellen, Schaffer, Jean E., Ory, Daniel S., Jiang, Xuntian

“…Niemann-Pick type C (NPC) disease is a rare lysosomal storage disorder caused by mutations in either the NPC1 or the NPC2 gene. A new class of lipids,…”
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Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1 by Yoon, Sea Young, Bagel, Jessica H, O'Donnell, Patricia A, Vite, Charles H, Wolfe, John H

“…Lysosomal storage diseases (LSDs) are debilitating neurometabolic disorders for most of which long-term effective therapies have not been developed. Gene…”
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Widespread correction of brain pathology in feline alpha-mannosidosis by dose escalation of intracisternal AAV vector injection by Hunter, Jacqueline E., Molony, Caitlyn M., Bagel, Jessica H., O’Donnell, Patricia, Vite, Charles H., Chawla, Sanjeev, Poptani, Harish, Wolfe, John H.

“…Alpha-mannosidosis is caused by a genetic deficiency of lysosomal alpha-mannosidase, leading to the widespread presence of storage lesions in the brain and…”
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Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors by Ferla, Rita, Alliegro, Marialuisa, Dell’Anno, Margherita, Nusco, Edoardo, Cullen, John M., Smith, Stephanie N., Wolfsberg, Tyra G., O’Donnell, Patricia, Wang, Ping, Nguyen, Anh-Dao, Chandler, Randy J., Chen, Zelin, Burgess, Shawn M., Vite, Charles H., Haskins, Mark E., Venditti, Charles P., Auricchio, Alberto

“…Adeno-associated viral (AAV) vectors have emerged as the preferred platform for in vivo gene transfer because of their combined efficacy and safety. However,…”
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Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna by Hinderer, Christian, Bell, Peter, Vite, Charles H, Louboutin, Jean-Pierre, Grant, Rebecca, Bote, Erin, Yu, Hongwei, Pukenas, Bryan, Hurst, Robert, Wilson, James M

“…Adeno-associated virus serotype 9 (AAV9) vectors have recently been shown to transduce cells throughout the central nervous system of nonhuman primates when…”
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Pulmonary abnormalities in animal models due to Niemann-Pick type C1 (NPC1) or C2 (NPC2) disease by Roszell, Blair R, Tao, Jian-Qin, Yu, Kevin J, Gao, Ling, Huang, Shaohui, Ning, Yue, Feinstein, Sheldon I, Vite, Charles H, Bates, Sandra R

“…Niemann-Pick C (NPC) disease is due to loss of NPC1 or NPC2 protein function that is required for unesterified cholesterol transport from the…”
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Identification of the rostral migratory stream in the canine and feline brain by Malik, Saafan Z, Lewis, Melissa, Isaacs, Alison, Haskins, Mark, Van Winkle, Thomas, Vite, Charles H, Watson, Deborah J

“…In the adult rodent brain, neural progenitor cells migrate from the subventricular zone of the lateral ventricle towards the olfactory bulb in a track known as…”
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Electrodiagnostic Testing and Histopathologic Changes Confirm Peripheral Nervous System Myelin Abnormalities in the Feline Model of Niemann-Pick Disease Type C by Bagel, Jessica H, Sikora, Tracey U, Prociuk, Maria, Pesayco, Jill P, Mizisin, Andrew P, Shelton, G Diane, Vite, Charles H

“…ABSTRACTNiemann-Pick disease type C (NPC disease) is an incurable, neurodegenerative, autosomal recessive disease caused by mutations in either the NPC1 or the…”
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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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