What Genetics Has Told Us and How It Can Inform Future Experiments for Spinal Muscular Atrophy, a Perspective

by Anton J. Blatnik, Vicki L. McGovern, Arthur H. M. Burghes
Published 2021-08-01

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

by Matthew E.R. Butchbach, Jonathan D. Edwards, Arthur H.M. Burghes
Published 2007-08-01

Ribonucleoprotein assembly defects correlate with spinal muscular atrophy severity and preferentially affect a subset of spliceosomal snRNPs.

by Francesca Gabanella, Matthew E R Butchbach, Luciano Saieva, Claudia Carissimi, Arthur H M Burghes, Livio Pellizzoni
Published 2007-09-01

Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic ∆7SMA mice

by Kaitlyn M. Kray, Vicki L. McGovern, Deepti Chugh, W. David Arnold, Arthur H.M. Burghes
Published 2021-11-01

Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy.

by Chalermchai Mitrpant, Paul Porensky, Haiyan Zhou, Loren Price, Francesco Muntoni, Sue Fletcher, Steve D Wilton, Arthur H M Burghes
Published 2013-01-01

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

by Vicki L McGovern, Aurélie Massoni-Laporte, Xueyong Wang, Thanh T Le, Hao T Le, Christine E Beattie, Mark M Rich, Arthur H M Burghes
Published 2015-01-01

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

by W David Arnold, Sandra Duque, Chitra C Iyer, Phillip Zaworski, Vicki L McGovern, Shannon J Taylor, Katharine M von Herrmann, Dione T Kobayashi, Karen S Chen, Stephen J Kolb, Sergey V Paushkin, Arthur H M Burghes
Published 2016-01-01