Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates
Meyer, Kathrin, Ferraiuolo, Laura, Schmelzer, Leah, Braun, Lyndsey, McGovern, Vicki, Likhite, Shibi, Michels, Olivia, Govoni, Alessandra, Fitzgerald, Julie, Morales, Pablo, Foust, Kevin D, Mendell, Jerry R, Burghes, Arthur H M, Kaspar, Brian K
Published in Molecular therapy (01.03.2015)
Published in Molecular therapy (01.03.2015)
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Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS
Foust, Kevin D, Salazar, Desirée L, Likhite, Shibi, Ferraiuolo, Laura, Ditsworth, Dara, Ilieva, Hristelina, Meyer, Kathrin, Schmelzer, Leah, Braun, Lyndsey, Cleveland, Don W, Kaspar, Brian K
Published in Molecular therapy (01.12.2013)
Published in Molecular therapy (01.12.2013)
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Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery
Bevan, Adam K., Hutchinson, Kirk R., Foust, Kevin D., Braun, Lyndsey, McGovern, Vicki L., Schmelzer, Leah, Ward, Jennifer G., Petruska, Jeffrey C., Lucchesi, Pamela A., Burghes, Arthur H.M., Kaspar, Brian K.
Published in Human molecular genetics (16.07.2010)
Published in Human molecular genetics (16.07.2010)
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Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model
Rodino-Klapac, Louise R, Janssen, Paul M L, Shontz, Kimberly M, Canan, Benjamin, Montgomery, Chrystal L, Griffin, Danielle, Heller, Kristin, Schmelzer, Leah, Handy, Chalonda, Clark, K Reed, Sahenk, Zarife, Mendell, Jerry R, Kaspar, Brian K
Published in Human molecular genetics (15.12.2013)
Published in Human molecular genetics (15.12.2013)
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Microglia Induce Motor Neuron Death via the Classical NF-κB Pathway in Amyotrophic Lateral Sclerosis
Frakes, Ashley E., Ferraiuolo, Laura, Haidet-Phillips, Amanda M., Schmelzer, Leah, Braun, Lyndsey, Miranda, Carlos J., Ladner, Katherine J., Bevan, Adam K., Foust, Kevin D., Godbout, Jonathan P., Popovich, Phillip G., Guttridge, Denis C., Kaspar, Brian K.
Published in Neuron (Cambridge, Mass.) (05.03.2014)
Published in Neuron (Cambridge, Mass.) (05.03.2014)
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A large animal model of spinal muscular atrophy and correction of phenotype
Duque, Sandra I., Arnold, W. David, Odermatt, Philipp, Li, Xiaohui, Porensky, Paul N., Schmelzer, Leah, Meyer, Kathrin, Kolb, Stephen J., Schümperli, Daniel, Kaspar, Brian K., Burghes, Arthur H. M.
Published in Annals of neurology (01.03.2015)
Published in Annals of neurology (01.03.2015)
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Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders
Bevan, Adam K, Duque, Sandra, Foust, Kevin D, Morales, Pablo R, Braun, Lyndsey, Schmelzer, Leah, Chan, Curtis M, McCrate, Mary, Chicoine, Louis G, Coley, Brian D, Porensky, Paul N, Kolb, Stephen J, Mendell, Jerry R, Burghes, Arthur HM, Kaspar, Brian K
Published in Molecular therapy (01.11.2011)
Published in Molecular therapy (01.11.2011)
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Electrophysiological biomarkers in spinal muscular atrophy: proof of concept
David Arnold, W., Porensky, Paul N., McGovern, Vicki L., Iyer, Chitra C., Duque, Sandra, Li, Xiaobai, Meyer, Kathrin, Schmelzer, Leah, Kaspar, Brian K., Kolb, Stephen J., Kissel, John T., Burghes, Arthur H. M.
Published in Annals of clinical and translational neurology (01.01.2014)
Published in Annals of clinical and translational neurology (01.01.2014)
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Journal Article
Microglia Induce Motor Neuron Death via the Classical NF-[kappa]B Pathway in Amyotrophic Lateral Sclerosis
Frakes, Ashley E, Ferraiuolo, Laura, Haidet-Phillips, Amanda M, Schmelzer, Leah, Braun, Lyndsey, Miranda, Carlos J, Ladner, Katherine J, Bevan, Adam K, Foust, Kevin D, Godbout, Jonathan P, Popovich, Phillip G, Guttridge, Denis C, Kaspar, Brian K
Published in Neuron (Cambridge, Mass.) (05.03.2014)
Published in Neuron (Cambridge, Mass.) (05.03.2014)
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Journal Article
Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept
Arnold, W David, Porensky, Paul N, McGovern, Vicki L, Iyer, Chitra C, Duque, Sandra, Li, Xiaobai, Meyer, Kathrin, Schmelzer, Leah, Kaspar, Brian K, Kolb, Stephen J, Kissel, John T, Burghes, Arthur H M
Published in Annals of clinical and translational neurology (01.01.2014)
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Published in Annals of clinical and translational neurology (01.01.2014)
Journal Article
Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery
Bevan, Adam K, Hutchinson, Kirk R, Foust, Kevin D, Braun, Lyndsey, McGovern, Vicki L, Schmelzer, Leah, Ward, Jennifer G, Petruska, Jeffrey C, Lucchesi, Pamela A, Burghes, Arthur H M, Kaspar, Brian K
Published in Human molecular genetics (15.10.2010)
Published in Human molecular genetics (15.10.2010)
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