Spinal Muscular Atrophy: Why do low levels of SMN make motor neurons sick?

Por Burghes, Arthur H.M., Beattie, Christine E.
Publicado em 2009

Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy

Por Porensky, Paul N., Burghes, Arthur H.M.
Publicado em 2013

Spinal Muscular Atrophy: The Development and Implementation of Potential Treatments Running Head: Spinal Muscular Atrophy

Por Arnold, W. David, Burghes, Arthur H.M.
Publicado em 2013

Antisense oligonucleotides and spinal muscular atrophy: skipping along

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

Abnormal motor phenotype in the SMNΔ7 mouse model of spinal muscular atrophy

Por Butchbach, Matthew E. R., Edwards, Jonathan D., Burghes, Arthur H. M.
Publicado em 2007

The effect of diet on the protective action of D156844 observed in spinal muscular atrophy mice

Por Butchbach, Matthew E. R., Singh, Jasbir, Gurney, Mark E., Burghes, Arthur H. M.
Publicado em 2014

Knockdown of the survival motor neuron (Smn) protein in zebrafish causes defects in motor axon outgrowth and pathfinding

Por McWhorter, Michelle L., Monani, Umrao R., Burghes, Arthur H.M., Beattie, Christine E.
Publicado em 2003

Identification of a Novel Cyclic AMP-response Element (CRE-II) and the Role of CREB-1 in the cAMP-induced Expression of the Survival Motor Neuron (SMN) Gene

Por Majumder, Sarmila, Varadharaj, Saradhadevi, Ghoshal, Kalpana, Monani, Umrao, Burghes, Arthur H. M., Jacob, Samson T.
Publicado em 2004

A novel method for oral delivery of drug compounds to the neonatal SMNΔ7 mouse model of spinal muscular atrophy

Por Butchbach, Matthew E. R., Edwards, Jonathan D., Schussler, Kristie R., Burghes, Arthur H. M.
Publicado em 2006

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

Ribonucleoprotein Assembly Defects Correlate with Spinal Muscular Atrophy Severity and Preferentially Affect a Subset of Spliceosomal snRNPs

Por Gabanella, Francesca, Butchbach, Matthew E. R., Saieva, Luciano, Carissimi, Claudia, Burghes, Arthur H. M., Pellizzoni, Livio
Publicado em 2007

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

Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy

Por Mitrpant, Chalermchai, Porensky, Paul, Zhou, Haiyan, Price, Loren, Muntoni, Francesco, Fletcher, Sue, Wilton, Steve D., Burghes, Arthur H. M.
Publicado em 2013

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

A SMN missense mutation complements SMN2 restoring snRNPs and rescuing SMA mice

Por Workman, Eileen, Saieva, Luciano, Carrel, Tessa L., Crawford, Thomas O., Liu, Don, Lutz, Cathleen, Beattie, Christine E., Pellizzoni, Livio, Burghes, Arthur H.M.
Publicado em 2009

A Positive Modifier of Spinal Muscular Atrophy in the SMN2 Gene

Por Prior, Thomas W., Krainer, Adrian R., Hua, Yimin, Swoboda, Kathryn J., Snyder, Pamela C., Bridgeman, Scott J., Burghes, Arthur H.M., Kissel, John T.
Publicado em 2009

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)

A genetic model of amyotrophic lateral sclerosis in zebrafish displays phenotypic hallmarks of motoneuron disease

Por Ramesh, Tennore, Lyon, Alison N., Pineda, Ricardo H., Wang, Chunping, Janssen, Paul M. L., Canan, Benjamin D., Burghes, Arthur H. M., Beattie, Christine E.
Publicado em 2010

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