Search Results - "Vera, Moin"

Immune-Mediated Inflammation May Contribute to the Pathogenesis of Cardiovascular Disease in Mucopolysaccharidosis Type I by Khalid, Omar, Vera, Moin U, Gordts, Philip L, Ellinwood, N Matthew, Schwartz, Philip H, Dickson, Patricia I, Esko, Jeffrey D, Wang, Raymond Y

“…Cardiovascular disease, a progressive manifestation of α-L-iduronidase deficiency or mucopolysaccharidosis type I, continues in patients both untreated and…”
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Combining angiotensin receptor blockade and enzyme replacement therapy for vascular disease in mucopolysaccharidosis type I by Hurt, Sarah C., Vera, Moin U., Le, Steven Q., Kan, Shih-hsin, Bui, Quang, Dickson, Patricia I.

“…Vascular involvement in the genetic disorder mucopolysaccharidosis type I (MPS I) has features of atherosclerotic disease near branch points of arterial…”
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Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs by Simonaro, Calogera M, Tomatsu, Shunji, Sikora, Tracy, Kubaski, Francyne, Frohbergh, Michael, Guevara, Johana M, Wang, Raymond Y, Vera, Moin, Kang, Jennifer L, Smith, Lachlan J, Schuchman, Edward H, Haskins, Mark E

“…We previously demonstrated the therapeutic benefits of pentosan polysulfate (PPS) in a rat model of mucopolysaccharidosis (MPS) type VI. Reduction of…”
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Evaluation of non-reducing end pathologic glycosaminoglycan detection method for monitoring therapeutic response to enzyme replacement therapy in human mucopolysaccharidosis I by Vera, Moin U., Le, Steven Q., Victoroff, Alla, Passage, Merry B., Brown, Jillian R., Crawford, Brett E., Polgreen, Lynda E., Chen, Agnes H., Dickson, Patricia I.

“…Therapeutic development and monitoring require demonstration of effects on disease phenotype. However, due to the complexity of measuring clinically-relevant…”
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Biochemical Studies in Fibroblasts to Interpret Variants of Unknown Significance in the ABCD1 Gene by van de Stadt, Stephanie I W, Mooyer, Petra A W, Dijkstra, Inge M E, Dekker, Conny J M, Vats, Divya, Vera, Moin, Ruzhnikov, Maura R Z, van Haren, Keith, Tang, Nelson, Koop, Klaas, Willemsen, Michel A, Hui, Joannie, Vaz, Frédéric M, Ebberink, Merel S, Engelen, Marc, Kemp, Stephan, Ferdinandusse, Sacha

“…Due to newborn screening for X-linked adrenoleukodystrophy (ALD), and the use of exome sequencing in clinical practice, the detection of variants of unknown…”
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Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I by Dickson, Patricia I., Ellinwood, N. Matthew, Brown, Jillian R., Witt, Robert G., Le, Steven Q., Passage, Merry B., Vera, Moin U., Crawford, Brett E.

“…Intrathecal enzyme replacement therapy is an experimental option to treat central nervous system disease due to lysosomal storage. Previous work shows that MPS…”
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A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I by Le, Steven Q, Kan, Shih-Hsin, Clarke, Don, Sanghez, Valentina, Egeland, Martin, Vondrak, Kristen N, Doherty, Terence M, Vera, Moin U, Iacovino, Michelina, Cooper, Jonathan D, Sands, Mark S, Dickson, Patricia I

“…Antibodies against recombinant proteins can significantly reduce their effectiveness in unanticipated ways. We evaluated the humoral response of mice with the…”
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IRF2BPL Is Associated with Neurological Phenotypes by Marcogliese, Paul C., Rosenfeld, Jill A., Koenig, Mary Kay, Chen, Agnes H., Dickson, Patricia I., Vera, Moin U., Salamon, Noriko, Infante, Elena, Poppe, Bruce, Chung, Hyung-Lok, Zuo, Zhongyuan, Kanca, Oguz, Xia, Fan, Smith, Edward C., Jasien, Joan, Kansagra, Sujay, Lark, Robert, Riley, Kacie, Golden-Grant, Katie, Poppe, Bruce, Terryn, Wim, Adams, David R., Allard, Patrick, Bacino, Carlos A., Bick, David P., Bostwick, Bret L., Brush, Matthew, Burrage, Lindsay C., Clark, Gary D., Cooper, Cynthia M., D’Souza, Precilla, Davids, Mariska, Dell’Angelica, Esteban C., Dhar, Shweta U., Dillon, Ani, Dorrani, Naghmeh, Dorset, Daniel C., Esteves, Cecilia, Fogel, Brent L., Gahl, William A., Gourdine, Jean-Philippe F., Haendel, Melissa, Hamid, Rizwan, Hanchard, Neil A., Howerton, Ellen M., Jiang, Yong-hui, Johnston, Jean M., Krier, Joel B., Lalani, Seema R., Lau, C. Christopher, Lee, Brendan H., Levy, Shawn E., Lewis, Richard A., Lipson, Allen, Loo, Sandra K., Macnamara, Ellen F., MacRae, Calum A., Manolio, Teri A., Martínez-Agosto, Julian A., McConkie-Rosell, Allyn, McCormack, Colleen E., Morimoto, Marie, Mulvihill, John J., Muzny, Donna M., Nelson, Stan F., Newberry, J. Scott, Nicholas, Sarah K., Pallais, J. Carl, Palmer, Christina G.S., Papp, Jeanette C., Parker, Neil H., Phillips, John A., Posey, Jennifer E., Postlethwait, John H., Potocki, Lorraine, Robertson, Amy K., Sampson, Jacinda B., Schoch, Kelly, Shashi, Vandana, Smith, Kevin S., Splinter, Kimberly, Stoler, Joan M., Sweetser, David A., Toro, Camilo, Urv, Tiina K., Vilain, Eric, Vogel, Tiphanie P., Wahl, Colleen E., Walley, Nicole M., Walsh, Chris A., Waters, Katrina M., Westerfield, Monte, Yu, Guoyun, Zheng, Allison, Yamamoto, Shinya, Wangler, Michael F., Lee, Brendan, Nelson, Stanley F., Goldstein, David B., Bellen, Hugo J.

“…Interferon regulatory factor 2 binding protein-like (IRF2BPL) encodes a member of the IRF2BP family of transcriptional regulators. Currently the biological…”
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IRF2BPL Is Associated with Neurological Phenotypes by Shashi, Vandana, Spillmann, Rebecca C., Stong, Nicholas, Rosenfeld, Jill A., Martínez-Agosto, Julián A., Herzog, Matthew, Chen, Agnes H., Dickson, Patricia I., Vera, Moin U., Graham, John M., Infante, Elena, Zuo, Zhongyuan, Lee, Pei-Tseng, Xia, Fan, Smith, Edward C., Jasien, Joan, Spiridigliozzi, Gail, Koeberl, Dwight D., Golden-Grant, Katie, Terryn, Wim, Adams, David R., Allard, Patrick, Bacino, Carlos A., Bick, David P., Bostwick, Bret L., Brush, Matthew, Burrage, Lindsay C., Clark, Gary D., Coakley, Terra R., Cooper, Cynthia M., D’Souza, Precilla, Davids, Mariska, Dell’Angelica, Esteban C., Dhar, Shweta U., Dillon, Ani, Dorrani, Naghmeh, Dorset, Daniel C., Douine, Emilie D., Esteves, Cecilia, Fogel, Brent L., Gahl, William A., Gourdine, Jean-Philippe F., Haendel, Melissa, Hamid, Rizwan, Hanchard, Neil A., Howerton, Ellen M., Jiang, Yong-hui, Johnston, Jean M., Krier, Joel B., Lalani, Seema R., Lau, C. Christopher, Lee, Brendan H., Levy, Shawn E., Lewis, Richard A., Lipson, Allen, Loo, Sandra K., Macnamara, Ellen F., MacRae, Calum A., Manolio, Teri A., Martínez-Agosto, Julian A., McConkie-Rosell, Allyn, McCormack, Colleen E., Morimoto, Marie, Mulvihill, John J., Muzny, Donna M., Nelson, Stan F., Newberry, J. Scott, Nicholas, Sarah K., Pallais, J. Carl, Palmer, Christina G.S., Papp, Jeanette C., Parker, Neil H., Phillips, John A., Posey, Jennifer E., Postlethwait, John H., Potocki, Lorraine, Robertson, Amy K., Sampson, Jacinda B., Samson, Susan L., Schoch, Kelly, Silverman, Edwin K., Smith, Kevin S., Splinter, Kimberly, Stoler, Joan M., Sweetser, David A., Toro, Camilo, Urv, Tiina K., Vilain, Eric, Vogel, Tiphanie P., Wahl, Colleen E., Walley, Nicole M., Walsh, Chris A., Waters, Katrina M., Westerfield, Monte, Yamamoto, Shinya, Yu, Guoyun, Zheng, Allison, Wangler, Michael F., Nelson, Stanley F., Bellen, Hugo J.

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Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients by Vera, Moin, Le, Steven, Kan, Shih-hsin, Garban, Hermes, Naylor, David, Mlikotic, Anton, Kaitila, Ilkka, Harmatz, Paul, Chen, Agnes, Dickson, Patricia

“…Background: Intrathecal (IT) enzyme replacement therapy with recombinant human α- L -iduronidase (rhIDU) has been studied to treat glycosaminoglycan storage in…”
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m.3685T > C is a novel mitochondrial DNA variant that causes Leigh syndrome by Jean, Jeffrey, Christodoulou, Eirini, Gai, Xiaowu, Tamrazi, Benita, Vera, Moin, Mitchell, Wendy G, Schmidt, Ryan J

“…Variants in the mitochondrial genome can result in dysfunction of Complex I within the electron transport chain, thus causing disruptions in oxidative…”
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430. Antibodies to Recombinant Human α-L-Iduronidase Enhance Uptake into Macrophages in Murine Mucopolysaccharidosis Type I by Sanghez, Valentina, Kan, Shih-Hsin, Le, Steven Q, Clarke, Don, Vondrak, Kristen, Vera, Moin U, Iacovino, Michelina, Sands, Mark S, Dickson, Patricia I

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Characterization on the cellular distribution of enzyme replacement therapy (ERT) under the influence of humoral immune response in MPS I mouse model by Sanghez, Valentina, Kan, Shih-hsin, Le, Steven Q, Clarke, Don, Vondrak, Kristen N, Vera, Moin, Iacovino, Michelina, Sands, Mark, Dickson, Patti

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Innate immune system activation in MPS I canine vascular disease by Vera, Moin, Le, Steven, Kan, Shih-hsin, Dickson, Patricia, Wang, Raymond Y.

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Pentosan polysulfate: New mechanistic insights and treatment of the mucopolysaccharidoses by Simonaro, Calogera M., Tomatsu, Shunji, Ge, Yi, Meng, Fanli, Frohbergh, Michael, Haskins, Mark E., Ruane, Therese, Wang, Raymond Y., Vera, Moin, Solyom, Alexander, Schuchman, Edward H.

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Aortic gene expression from the canine model of MPS I identifies upregulation of genes related to antigen presentation and inflammatory cytokines, and downregulation of cellular adhesion and cytoskeletal genes by Wang, Raymond Y., Vera, Moin, Khalid, Omar, Ellinwood, N. Matthew, Schwartz, Phillip, Dickson, Patricia, Gordts, Phillip, Esko, Jeff

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The humoral immune response to intravenous recombinant human alpha-L-iduronidase alters its tissue delivery in murine mucopolysaccharidosis I by Dickson, Patricia, Le, Steven Q., Kan, Shih-hsin, Vera, Moin U.

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Immune response to intrathecal enzyme therapy in Mucopolysaccharidosis I patients by Vera, Moin, Le, Steven, Garban, Hermes, Orchard, Paul, Tolar, Jakub, Dickson, Patricia

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Immune-Mediated Inflammation May Contribute to the Pathogenesis of Cardiovascular Disease in Mucopolysaccharidosis Type I: e0150850 by Khalid, Omar, Vera, Moin U, Gordts, Philip L, Ellinwood, N Matthew, Schwartz, Philip H, Dickson, Patricia I, Esko, Jeffrey D, Wang, Raymond Y

“…Background Cardiovascular disease, a progressive manifestation of [alpha] -L-iduronidase deficiency or mucopolysaccharidosis type I, continues in patients both…”
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Mannose 6-Phosphate Conjugation Is Not Sufficient to Allow Induction of Immune Tolerance to Phenylalanine Ammonia-Lyase in Dogs by Vera, Moin, Lester, Thomas, Zhao, Bin, Tiger, Pascale, Clarke, Scott, Tippin, Brigette L., Passage, Merry B., Le, Steven Q., Femenia, Javier, Lemontt, Jeffrey F., Kakkis, Emil D., Dickson, Patricia I.

“…The immune response to exogenous protein has been shown to reduce therapeutic efficacy in animal models of enzyme replacement therapy. A previously published…”
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