Rational design of antisense oligomers to induce dystrophin exon skipping

by Chalermchai Mitrpant, Abbie M. Adams, Penny L. Meloni, Francesco Muntoni, Sue Fletcher, Steve D. Wilton
Published 2018

Histopathological defects in intestine in severe spinal muscular atrophy mice are improved by systemic antisense oligonucleotide treatment

by Palittiya Sintusek, Francesco Catapano, Napat Angkathunkayul, Elena Marrosu, Simon H. Parson, Jennifer E. Morgan, Francesco Muntoni, Haiyan Zhou
Published 2018

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

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