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 2018

Complement-mediated muscle cell lysis: A possible mechanism of myonecrosis in anti-SRP associated necrotizing myopathy (ASANM)

by Arada Rojana-udomsart, Chalermchai Mitrpant, Christine Bundell, Loren Price, Yue Bei Luo, Victoria Fabian, Steve D. Wilton, Peter Hollingsworth, Frank L. Mastaglia
Published 2018

A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice

by Haiyan Zhou, Narinder Janghra, Chalermchai Mitrpant, Rachel L. Dickinson, Karen Anthony, Loren Price, Ian C. Eperon, Stephen D. Wilton, Jennifer Morgan, Francesco Muntoni
Published 2018