Antisense oligonucleotides and spinal muscular atrophy: skipping along

Por Burghes, Arthur H.M., McGovern, Vicki L.
Publicado em 2010

Embryonic motor axon development in the severe SMA mouse

Por McGovern, Vicki L., Gavrilina, Tatiana O., Beattie, Christine E., Burghes, Arthur H.M.
Publicado em 2008

Deletion of atrophy enhancing genes fails to ameliorate the phenotype in a mouse model of spinal muscular atrophy

Por Iyer, Chitra C., McGovern, Vicki L., Wise, Dawnne O., Glass, David J., Burghes, Arthur H. M.
Publicado em 2014

Temporal requirement for high SMN expression in SMA mice

Por Le, Thanh T., McGovern, Vicki L., Alwine, Isaac E., Wang, Xueyong, Massoni-Laporte, Aurelie, Rich, Mark M., Burghes, Arthur H.M.
Publicado em 2011

Plastin 3 Expression Does Not Modify Spinal Muscular Atrophy Severity in the ∆7 SMA Mouse

Por McGovern, Vicki L., Massoni-Laporte, Aurélie, Wang, Xueyong, Le, Thanh T., Le, Hao T., Beattie, Christine E., Rich, Mark M., Burghes, Arthur H. M.
Publicado no PLoS One (2015)

The neuromuscular impact of symptomatic SMN restoration in a mouse model of spinal muscular atrophy

Por Arnold, W. David, McGovern, Vicki L., Sanchez, Benjamin, Li, Jia, Corlett, Kaitlyn M., Kolb, Stephen J., Rutkove, Seward B., Burghes, Arthur H.
Publicado no Neurobiol Dis (2015)

A Role for SMN Exon 7 Splicing in the Selective Vulnerability of Motor Neurons in Spinal Muscular Atrophy

Por Ruggiu, Matteo, McGovern, Vicki L., Lotti, Francesco, Saieva, Luciano, Li, Darrick K., Kariya, Shingo, Monani, Umrao R., Burghes, Arthur H. M., Pellizzoni, Livio
Publicado em 2012

A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

Por Porensky, Paul N., Mitrpant, Chalermchai, McGovern, Vicki L., Bevan, Adam K., Foust, Kevin D., Kaspar, Brain K., Wilton, Stephen D., Burghes, Arthur H.M.
Publicado em 2012

SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA

Por McGovern, Vicki L., Iyer, Chitra C., Arnold, W. David, Gombash, Sara E., Zaworski, Phillip G., Blatnik, Anton J., Foust, Kevin D., Burghes, Arthur H.M.
Publicado no Hum Mol Genet (2015)

Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNΔ7 mouse model of SMA

Por Iyer, Chitra C., McGovern, Vicki L., Murray, Jason D., Gombash, Sara E., Zaworski, Phillip G., Foust, Kevin D., Janssen, Paul M.L., Burghes, Arthur H.M.
Publicado no Hum Mol Genet (2015)

Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

Por Gavrilina, Tatiana O., McGovern, Vicki L., Workman, Eileen, Crawford, Thomas O., Gogliotti, Rocky G., DiDonato, Christine J., Monani, Umrao R., Morris, Glenn E., Burghes, Arthur H.M.
Publicado em 2008

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Por Foust, Kevin D, Wang, Xueyong, McGovern, Vicki L, Braun, Lyndsey, Bevan, Adam K, Haidet, Amanda M, Le, Thanh T, Morales, Pablo R, Rich, Mark M, Burghes, Arthur H M, Kaspar, Brian K
Publicado em 2010

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)

SMN deficiency disrupts gastrointestinal and enteric nervous system function in mice

Por Gombash, Sara E., Cowley, Christopher J., Fitzgerald, Julie A., Iyer, Chitra C., Fried, David, McGovern, Vicki L., Williams, Kent C., Burghes, Arthur H.M., Christofi, Fedias L., Gulbransen, Brian D., Foust, Kevin D.
Publicado no Hum Mol Genet (2015)

SMN deficiency disrupts gastrointestinal and enteric nervous system function in mice

Por Gombash, Sara E., Cowley, Christopher J., Fitzgerald, Julie A., Iyer, Chitra C., Fried, David, McGovern, Vicki L., Williams, Kent C., Burghes, Arthur H.M., Christofi, Fedias L., Gulbransen, Brian D., Foust, Kevin D.
Publicado no Hum Mol Genet (2015)

Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration

Por Arnold, W. David, Duque, Sandra, Iyer, Chitra C., Zaworski, Phillip, McGovern, Vicki L., Taylor, Shannon J., von Herrmann, Katharine M., Kobayashi, Dione T., Chen, Karen S., Kolb, Stephen J., Paushkin, Sergey V., Burghes, Arthur H. M.
Publicado no PLoS One (2016)

Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study

Por Kolb, Stephen J., Coffey, Christopher S., Yankey, Jon W., Krosschell, Kristin, Arnold, W. David, Rutkove, Seward B., Swoboda, Kathryn J., Reyna, Sandra P., Sakonju, Ai, Darras, Basil T., Shell, Richard, Kuntz, Nancy, Castro, Diana, Iannaccone, Susan T., Parsons, Julie, Connolly, Anne M., Chiriboga, Claudia A., McDonald, Craig, Burnette, W. Bryan, Werner, Klaus, Thangarajh, Mathula, Shieh, Perry B., Finanger, Erika, Cudkowicz, Merit E., McGovern, Michelle M., McNeil, D. Elizabeth, Finkel, Richard, Kaye, Edward, Kingsley, Allison, Renusch, Samantha R., McGovern, Vicki L., Wang, Xueqian, Zaworski, Phillip G., Prior, Thomas W., Burghes, Arthur H.M., Bartlett, Amy, Kissel, John T.
Publicado no Ann Clin Transl Neurol (2016)