Search Results - "Hinderer, Christian J."

Overcoming preexisting humoral immunity to AAV using capsid decoys by Mingozzi, Federico, Anguela, Xavier M, Pavani, Giulia, Chen, Yifeng, Davidson, Robert J, Hui, Daniel J, Yazicioglu, Mustafa, Elkouby, Liron, Hinderer, Christian J, Faella, Armida, Howard, Carolann, Tai, Alex, Podsakoff, Gregory M, Zhou, Shangzhen, Basner-Tschakarjan, Etiena, Wright, John Fraser, High, Katherine A

“…Adeno-associated virus (AAV) vectors delivered through the systemic circulation successfully transduce various target tissues in animal models. However,…”
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Adeno‐associated virus‐vectored erythropoietin gene therapy for anemia in cats with chronic kidney disease by Vaden, Shelly L., Kendall, Allison R., Foster, Jonathan D., New, Heidi L., Eagleson, Jane S., May, Jacky L., Traas, Anne M., Wilson, Matthew J., McIntyre, Beth H., Hinderer, Christian J., Olenick, Lauren K., Wilson, James M.

“…Background A treatment of chronic kidney disease (CKD)‐associated anemia in cats is needed. SB‐001 is an adeno‐associated virus‐vectored (AAV)‐based gene…”
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Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy by Rojsajjakul, Teerapat, Hordeaux, Juliette J., Choudhury, Gourav R., Hinderer, Christian J., Mesaros, Clementina, Wilson, James M., Blair, Ian A.

“…Deficiency in human mature frataxin (hFXN-M) protein is responsible for the devastating neurodegenerative and cardiodegenerative disease of Friedreich’s ataxia…”
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Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors by Horiuchi, Makoto, Hinderer, Christian J., Greig, Jenny A., Dyer, Cecilia, Buza, Elizabeth L., Bell, Peter, Chichester, Jessica A., Hayashi, Peter M., Yan, Hanying, Goode, Tamara, Wilson, James M.

“…Gene therapy using neurotropic adeno-associated virus vectors represents an emerging solution for genetic disorders affecting the central nervous system. The…”
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Validation of high-sensitivity assays to quantitate cerebrospinal fluid and serum β-galactosidase activity in patients with GM1-gangliosidosis by Quadrini, Karen J., Vrentas, Catherine, Duke, Christian, Wilson, Chris, Hinderer, Christian J., Weinstein, David A., Al-Zaidy, Samiah A., Browne, Susan E., Wilson, James M., Ni, Yan G.

“…GM1-gangliosidosis (GM1) is a lysosomal storage disorder caused by mutations in the galactosidase beta 1 gene (GLB1) that leads to reduced β-galactosidase…”
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Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors by Horiuchi, Makoto, Hinderer, Christian J., Greig, Jenny A., Dyer, Cecilia, Buza, Elizabeth L., Bell, Peter, Chichester, Jessica A., Hayashi, Peter M., Yan, Hanying, Goode, Tamara, Wilson, James M.

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Erratum: Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors by Horiuchi, Makoto, Hinderer, Christian J, Greig, Jenny A, Dyer, Cecilia, Buza, Elizabeth L, Bell, Peter, Chichester, Jessica A, Hayashi, Peter M, Yan, Hanying, Goode, Tamara, Wilson, James M

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A Novel Strategy to Circumvent Pre-Existing Humoral Immunity to AAV by Mingozzi, Federico, Anguela, Xavier M, Pavani, Giulia, Chen, Yifeng, Davidson, Robert J, Hui, Daniel J., Hinderer, Christian J, Faella, Armida, Howard, Crolann, Tai, Alex, Podsakoff, Gregory M, Ragni, Margaret V., Zhou, Shangzhen, Basner-Tschakarjan, Etiena, Wright, J. Fraser, High, Katherine A.

“…Abstract 2050 Adeno-associated viral (AAV) vector-mediated gene transfer has shown great potential as a therapeutic platform for inherited and metabolic…”
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