Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS

Por 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
Publicado em 2013

A large animal model of Spinal Muscular Atrophy and correction of phenotype

Por 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.
Publicado no Ann Neurol (2015)

Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

Por 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.
Publicado em 2010

Electrophysiological biomarkers in spinal muscular atrophy: proof of concept

Por 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
Publicado em 2014

Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

Por 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
Publicado no Mol Ther (2015)

Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model

Por 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.
Publicado em 2013

Microglia induce motor neuron death via the classical NF-κB pathway in amyotrophic lateral sclerosis

Por 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.
Publicado em 2014

Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

Por 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
Publicado em 2011