Myotonic dystrophy: Therapeutic strategies for the future

Por Wheeler, Thurman M.
Publicado no Neurotherapeutics (2008)

RNA Interference Targeting CUG Repeats in a Mouse Model of Myotonic Dystrophy

Por Sobczak, Krzysztof, Wheeler, Thurman M, Wang, Wenli, Thornton, Charles A
Publicado em 2013

Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy

Por Wheeler, Thurman M., Lueck, John D., Swanson, Maurice S., Dirksen, Robert T., Thornton, Charles A.
Publicado em 2007

Progressive myopathy in an inducible mouse model of oculopharyngeal muscular dystrophy

Por Mankodi, Ami, Wheeler, Thurman M., Shetty, Reena, Salceies, Kelly M., Becher, Mark W., Thornton, Charles A.
Publicado em 2011

Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration

Por Bertoni, Carmen, Jarrahian, Sohail, Wheeler, Thurman M., Li, Yining, Olivares, Eric C., Calos, Michele P., Rando, Thomas A.
Publicado em 2006

Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy

Por Kanadia, Rahul N., Shin, Jihae, Yuan, Yuan, Beattie, Stuart G., Wheeler, Thurman M., Thornton, Charles A., Swanson, Maurice S.
Publicado em 2006

Reversal of RNA-dominance by displacement of protein sequestered on triplet repeat RNA

Por Wheeler, Thurman M., Sobczak, Krzysztof, Lueck, John D., Osborne, Robert J., Lin, Xiaoyan, Dirksen, Robert T., Thornton, Charles A.
Publicado em 2009

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Por Wheeler, Thurman M., Leger, Andrew J., Pandey, Sanjay K., MacLeod, A. Robert, Nakamori, Masayuki, Cheng, Seng H., Wentworth, Bruce M., Bennett, C. Frank, Thornton, Charles A.
Publicado no Nature (2012)

Identification and Characterization of Modified Antisense Oligonucleotides Targeting DMPK in Mice and Nonhuman Primates for the Treatment of Myotonic Dystrophy Type 1

Por Pandey, Sanjay K., Wheeler, Thurman M., Justice, Samantha L., Kim, Aneeza, Younis, Husam S., Gattis, Danielle, Jauvin, Dominic, Puymirat, Jack, Swayze, Eric E., Freier, Susan M., Bennett, C. Frank, Thornton, Charles A., MacLeod, A. Robert
Publicado no J Pharmacol Exp Ther (2015)

Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

Por Mulders, Susan A. M., van den Broek, Walther J. A. A., Wheeler, Thurman M., Croes, Huib J. E., van Kuik-Romeijn, Petra, de Kimpe, Sjef J., Furling, Denis, Platenburg, Gerard J., Gourdon, Geneviève, Thornton, Charles A., Wieringa, Bé, Wansink, Derick G.
Publicado em 2009

Splicing biomarkers of disease severity in myotonic dystrophy

Por Nakamori, Masayuki, Sobczak, Krzysztof, Puwanant, Araya, Welle, Steve, Eichinger, Katy, Pandya, Shree, Dekdebrun, Jeannne, Heatwole, Chad R., McDermott, Michael P., Chen, Tian, Cline, Melissa, Tawil, Rabi, Osborne, Robert J., Wheeler, Thurman M., Swanson, Maurice, Moxley, Richard T., Thornton, Charles A.
Publicado em 2013