Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2 … S Cirak, V Arechavala-Gomeza, M Guglieri, L Feng, S Torelli, K Anthony, ... The Lancet 378 (9791), 595-605, 2011 | 1165 | 2011 |
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of … M Kinali, V Arechavala-Gomeza, L Feng, S Cirak, D Hunt, C Adkin, ... The Lancet Neurology 8 (10), 918-928, 2009 | 882 | 2009 |
ALS genetics, mechanisms, and therapeutics: where are we now? R Mejzini, LL Flynn, IL Pitout, S Fletcher, SD Wilton, PA Akkari Frontiers in neuroscience 13, 1310, 2019 | 743 | 2019 |
Regulation of eukaryotic gene expression by the untranslated gene regions and other non-coding elements LW Barrett, S Fletcher, SD Wilton Cellular and molecular life sciences 69, 3613-3634, 2012 | 710 | 2012 |
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology J Alter, F Lou, A Rabinowitz, HF Yin, J Rosenfeld, SD Wilton, TA Partridge, ... Nature medicine 12 (2), 175-177, 2006 | 709 | 2006 |
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse QL Lu, CJ Mann, F Lou, G Bou-Gharios, GE Morris, S Xue, S Fletcher, ... Nature medicine 9 (8), 1009-1014, 2003 | 593 | 2003 |
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse CJ Mann, K Honeyman, AJ Cheng, T Ly, F Lloyd, S Fletcher, JE Morgan, ... Proceedings of the National Academy of Sciences 98 (1), 42-47, 2001 | 542 | 2001 |
A mutation in the α tropomyosin gene TPM3 associated with autosomal dominant nemaline myopathy NG Laing, SD Wilton, PA Akkari, S Dorosz, K Boundy, C Kneebone, ... Nature genetics 9 (1), 75-79, 1995 | 429 | 1995 |
Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript SD Wilton, AM Fall, PL Harding, G McClorey, C Coleman, S Fletcher Molecular Therapy 15 (7), 1288-1296, 2007 | 372 | 2007 |
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD G McClorey, HM Moulton, PL Iversen, S Fletcher, SD Wilton Gene therapy 13 (19), 1373-1381, 2006 | 363 | 2006 |
Detection and identification of multiple mycobacterial pathogens by DNA amplification in a single tube. S Wilton, D Cousins Genome Research 1 (4), 269-273, 1992 | 363 | 1992 |
Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides SD Wilton, F Lloyd, K Carville, S Fletcher, K Honeyman, S Agrawal, ... Neuromuscular disorders 9 (5), 330-338, 1999 | 356 | 1999 |
Mutations in the nebulin gene associated with autosomal recessive nemaline myopathy K Pelin, P Hilpelä, K Donner, C Sewry, PA Akkari, SD Wilton, ... Proceedings of the National Academy of Sciences 96 (5), 2305-2310, 1999 | 353 | 1999 |
A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse PN Porensky, C Mitrpant, VL McGovern, AK Bevan, KD Foust, BK Kaspar, ... Human molecular genetics 21 (7), 1625-1638, 2012 | 315 | 2012 |
Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle BL Gebski, CJ Mann, S Fletcher, SD Wilton Human molecular genetics 12 (15), 1801-1811, 2003 | 282 | 2003 |
Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy CJ Mann, K Honeyman, G McClorey, S Fletcher, SD Wilton The Journal of Gene Medicine: A cross‐disciplinary journal for research on …, 2002 | 279 | 2002 |
Use of polymerase chain reaction for rapid diagnosis of tuberculosis DV Cousins, SD Wilton, BR Francis, BL Gow Journal of Clinical Microbiology 30 (1), 255-258, 1992 | 278 | 1992 |
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD C Pichavant, A Aartsma-Rus, PR Clemens, KE Davies, G Dickson, ... Molecular Therapy 19 (5), 830-840, 2011 | 274 | 2011 |
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide S Fletcher, K Honeyman, AM Fall, PL Harding, RD Johnsen, SD Wilton The Journal of Gene Medicine: A cross‐disciplinary journal for research on …, 2006 | 270 | 2006 |
Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle V Arechavala-Gomeza, IR Graham, LJ Popplewell, AM Adams, ... Human gene therapy 18 (9), 798-810, 2007 | 253 | 2007 |