Personalised genetic intervention for duchenne muscular dystrophy: Antisense oligomers and exon skipping

by Chalermchai Mitrpant, Sue Fletcher, Steve D. Wilton
Published 2018

By-passing the nonsense mutation in the 4^CV mouse model of muscular dystrophy by induced exon skipping

by Chalermchai Mitrpant, Sue Fletcher, Patrick L. Iversen, Steve D. Wilton
Published 2018

Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript

by Loren L. Flynn, Chalermchai Mitrpant, Ianthe L. Pitout, Sue Fletcher, Steve D. Wilton
Published 2019

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

Targeted SMN exon skipping: A useful control to assess in vitro and in vivo splice-switching studies

by Loren L. Flynn, Chalermchai Mitrpant, Abbie Adams, Ianthe L. Pitout, Anja Stirnweiss, Sue Fletcher, Steve D. Wilton
Published 2022

Consequences of Making the Inactive Active Through Changes in Antisense Oligonucleotide Chemistries

by Khine Zaw, Kane Greer, May Thandar Aung-Htut, Chalermchai Mitrpant, Rakesh N. Veedu, Sue Fletcher, Steve D. Wilton
Published 2020

Antisense oligonucleotide induction of progerin in human myogenic cells

by Yue Bei Luo, Chalermchai Mitrpant, Abbie M. Adams, Russell D. Johnsen, Sue Fletcher, Frank L. Mastaglia, Steve D. Wilton
Published 2018

Investigation of splicing changes and post-translational processing of LMNA in sporadic inclusion body myositis

by Yue Bei Luo, Chalermchai Mitrpant, Russell Johnsen, Vicki Fabian, Merrilee Needham, Sue Fletcher, Steve D. Wilton, Frank L. Mastaglia
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

Investigation of age-related changes in LMNA splicing and expression of progerin in human skeletal muscles

by Yue Bei Luo, Chalermchai Mitrpant, Russell D. Johnsen, Victoria A. Fabian, Sue Fletcher, Frank L. Mastaglia, Steve D. Wilton
Published 2018

Induction of cryptic pre-mRNA splice-switching by antisense oligonucleotides

by Kristin A. Ham, Niall P. Keegan, Craig S. McIntosh, May T. Aung-Htut, Khine Zaw, Kane Greer, Sue Fletcher, Steve D. Wilton
Published 2022

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

Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts

by Jarichad Toosaranont, Sukanya Ruschadaariyachat, Warasinee Mujchariyakul, Jantarika Kumar Arora, Varodom Charoensawan, Bhoom Suktitipat, Thomas N. Palmer, Sue Fletcher, Steve D. Wilton, Chalermchai Mitrpant
Published 2022

Analysis of HLA-DRB3 alleles and supertypical genotypes in the MHC Class II region in sporadic inclusion body myositis

by Arada Rojana-Udomsart, Chalermchai Mitrpant, Ian James, Campbell Witt, Merrilee Needham, Timothy Day, Lynette Kiers, Alastair Corbett, Patricia Martinez, Steve D. Wilton, Frank L. Mastaglia
Published 2018

Generation of three induced pluripotent stem cell lines from a patient with Usher syndrome caused by biallelic c.949C > A and c.1256G > T mutations in the USH2A gene

by Khine Zaw, Elaine Y.M. Wong, Xiao Zhang, Dan Zhang, Shang Chih Chen, Jennifer A. Thompson, Tina Lamey, Terri McLaren, John N. De Roach, Steve D. Wilton, Sue Fletcher, Chalermchai Mitrpant, Marcus D. Atlas, Fred K. Chen, Samuel McLenachan
Published 2022