How will the field of gene therapy survive its success? von Kaemmerer, William F.

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Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay–Sachs Disease and Sandhoff Disease Mouse Models von Kot, Shalini, Karumuthil-Melethil, Subha, Woodley, Evan, Zaric, Violeta, Thompson, Patrick, Chen, Zhilin, Lykken, Erik, Keimel, John G., Kaemmerer, William F., Gray, Steven J., Walia, Jagdeep S.

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Intracerebroventricular amyloid-β antibodies reduce cerebral amyloid angiopathy and associated micro-hemorrhages in aged Tg2576 mice von Thakker, Deepak R, Weatherspoon, Marcy R, Harrison, Jonathan, Keene, Thomas E, Lane, Deanna S, Kaemmerer, William F, Stewart, Gregory R, Shafer, Lisa L

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Onset Time and Durability of Huntingtin Suppression in Rhesus Putamen After Direct Infusion of Antihuntingtin siRNA von Grondin, Richard, Ge, Pei, Chen, Qingmin, Sutherland, Jessica E, Zhang, Zhiming, Gash, Don M, Stiles, David K, Stewart, Gregory R, Sah, Dinah W Y, Kaemmerer, William F

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The effects of huntingtin-lowering: what do we know so far? von Kaemmerer, William F, Grondin, Richard C

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Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum von GRONDIN, Richard, KAYTOR, Michael D, KAEMMERER, William F, YI AI, NELSON, Peter T, THAKKER, Deepak R, HEISEL, Jennifer, WEATHERSPOON, Marcy R, BLUM, Janelle L, BURRIGHT, Eric N, ZHIMING ZHANG

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A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference von Lombardi, Maria Stella, Jaspers, Leonie, Spronkmans, Christine, Gellera, Cinzia, Taroni, Franco, Di Maria, Emilio, Donato, Stefano Di, Kaemmerer, William F.

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Widespread suppression of huntingtin with convection-enhanced delivery of siRNA von Stiles, David K., Zhang, Zhiming, Ge, Pei, Nelson, Brian, Grondin, Richard, Ai, Yi, Hardy, Peter, Nelson, Peter T., Guzaev, Andrei P., Butt, Mark T., Charisse, Klaus, Kosovrasti, Verbena, Tchangov, Lubomir, Meys, Michael, Maier, Martin, Nechev, Lubomir, Manoharan, Muthiah, Kaemmerer, William F., Gwost, Douglas, Stewart, Gregory R., Gash, Don M., Sah, Dinah W.Y.

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In Vivo Transduction of Cerebellar Purkinje Cells Using Adeno-Associated Virus Vectors von Kaemmerer, William F., Reddy, Rukmini G., Warlick, Christopher A., Hartung, Seth D., McIvor, R. Scott, Low, Walter C.

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Adeno-associated virus type 2 vectors: transduction and long-term expression in cerebellar Purkinje cells in vivo is mediated by the fibroblast growth factor receptor 1: bFGFR-1 me... von Belur, Lalitha R., Kaemmerer, William F., McIvor, R. Scott, Low, Walter C.

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87 Intrathecal continuous or intermittent bolus delivery of laronidase results in elevated levels in canine CNS tissue von Keimel, John, Keimel, John G., Passage, Merry, Billstrom, Tina, Lentz, Linnea, Rose, Rebecca, Kaemmerer, William F.

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Distribution of Patients′ Paroxysmal Atrial Tachyarrhythmia Episodes: Implications for Detection of Treatment Efficacy von KAEMMERER, WILLIAM F., ROSE, M. SARAH, MEHRA, RAHUL

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Adeno-associated virus type 2 vectors: transduction and long-term expression in cerebellar Purkinje cells in vivo is mediated by the fibroblast growth factor receptor 1 von Belur, Lalitha R, Kaemmerer, William F, McIvor, R. Scott, Low, Walter C

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Measuring the perceived support for innovation in organizations von Siegel, Saul M, Kaemmerer, William F

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Intracerebroventricular amyloid-[beta] antibodies reduce cerebral amyloid angiopathy and associated micro-hemorrhages in aged Tg2576 mice von Thakker, Deepak R, Weatherspoon, Marcy R, Harrison, Jonathan, Keene, Thomas E, Lane, Deanna S, Kaemmerer, William F, Stewart, Gregory R, Shafer, Lisa L

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Intracerebroventricular amyloid-β antibodies reduce cerebral amyloid angiopathy and associated micro-hemorrhages in aged Tg2576 mice von Thakker, Deepak R, Weatherspoon, Marcy R, Harrison, Jonathan, Keene, Thomas E, Lane, Deanna S, Kaemmerer, William F, Stewart, Gregory R, Shafer, Lisa L

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Cerebellar Allografts Survive and Transiently Alleviate Ataxia in a Transgenic Model of Spinocerebellar Ataxia Type-1 von Kaemmerer, William F., Low, Walter C.

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WITHDRAWN: 32 Intrathecal continuous or intermittent bolus delivery of laronidase results in elevated levels in canine CNS tissue von Keimel, John, Keimel, John G., Lentz, Linnea, Passage, Merry, Rose, Rebecca, Billstrom, Tina, Kaemmerer, William F.

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Therapeutic window determination von Kaemmerer, William F

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Electrode selection for electrical stimulation therapy von Connolly, Allison T, Kaemmerer, William F, Dani, Siddharth

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