A Negatively Acting Bifunctional RNA Increases Survival Motor Neuron Both In Vitro and In Vivo

Por Dickson, Alexa, Osman, Erkan, Lorson, Christian L.
Publicado em 2008

Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular Atrophy

Por Osman, Erkan Y, Yen, Pei-Fen, Lorson, Christian L
Publicado em 2012

Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy

Por Baughan, Travis D., Dickson, Alexa, Osman, Erkan Y., Lorson, Christian L.
Publicado em 2009

Defining the therapeutic window in a severe animal model of spinal muscular atrophy

Por Robbins, Kate L., Glascock, Jacqueline J., Osman, Erkan Y., Miller, Madeline R., Lorson, Christian L.
Publicado em 2014

Delivery of Therapeutic Agents Through Intracerebroventricular (ICV) and Intravenous (IV) Injection in Mice

Por Glascock, Jacqueline J., Osman, Erkan Y., Coady, Tristan H., Rose, Ferrill F., Shababi, Monir, Lorson, Christian L.
Publicado em 2011

Decreasing Disease Severity in Symptomatic, Smn(−/−);SMN2(+/+), Spinal Muscular Atrophy Mice Following scAAV9-SMN Delivery

Por Glascock, Jacqueline J., Osman, Erkan Y., Wetz, Mary J., Krogman, Megan M., Shababi, Monir, Lorson, Christian L.
Publicado em 2012

Placental Development in a Mouse Model of Spinal Muscular Atrophy

Por Van Granigen Caesar, Gerialisa, Dale, Jeffrey M., Osman, Erkan Y., Garcia, Michael L., Lorson, Christian L., Schulz, Laura C.
Publicado no Biochem Biophys Res Commun (2015)

Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models

Por Osman, Erkan Y., Miller, Madeline R., Robbins, Kate L., Lombardi, Abby M., Atkinson, Arleigh K., Brehm, Amanda J., Lorson, Christian L.
Publicado em 2014

Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy

Por Kaifer, Kevin A., Villalón, Eric, Osman, Erkan Y., Glascock, Jacqueline J., Arnold, Laura L., Cornelison, D.D.W., Lorson, Christian L.
Publicado no JCI Insight (2017)

Comparison of independent screens on differentially vulnerable motor neurons reveals alpha-synuclein as a common modifier in motor neuron diseases

Por Kline, Rachel A., Kaifer, Kevin A., Osman, Erkan Y., Carella, Francesco, Tiberi, Ariana, Ross, Jolill, Pennetta, Giuseppa, Lorson, Christian L., Murray, Lyndsay M.
Publicado no PLoS Genet (2017)

Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds

Por Cherry, Jonathan J, Osman, Erkan Y, Evans, Matthew C, Choi, Sungwoon, Xing, Xuechao, Cuny, Gregory D, Glicksman, Marcie A, Lorson, Christian L, Androphy, Elliot J
Publicado em 2013

Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy

Por Osman, Erkan Y, Washington, Charles W, Kaifer, Kevin A, Mazzasette, Chiara, Patitucci, Teresa N, Florea, Kyra M, Simon, Madeline E, Ko, Chien-Ping, Ebert, Allison D, Lorson, Christian L
Publicado no Mol Ther (2016)

Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent

Por Shababi, Monir, Feng, Zhihua, Villalon, Eric, Sibigtroth, Christine M, Osman, Erkan Y, Miller, Madeline R, Williams-Simon, Patricka A, Lombardi, Abby, Sass, Thalia H, Atkinson, Arleigh K, Garcia, Michael L, Ko, Chien-Ping, Lorson, Christian L
Publicado no Mol Ther (2016)