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, 2019 | 716 | 2019 |
Identification of IL6R and chromosome 11q13. 5 as risk loci for asthma MAR Ferreira, MC Matheson, DL Duffy, GB Marks, J Hui, P Le Souëf, ... The Lancet 378 (9795), 1006-1014, 2011 | 469 | 2011 |
Meta-analysis of genome-wide association studies identifies ten loci influencing allergic sensitization K Bønnelykke, MC Matheson, TH Pers, R Granell, DP Strachan, AC Alves, ... Nature genetics 45 (8), 902-906, 2013 | 293 | 2013 |
Genome-wide association analysis identifies 11 risk variants associated with the asthma with hay fever phenotype MAR Ferreira, MC Matheson, CS Tang, R Granell, W Ang, J Hui, AK Kiefer, ... Journal of Allergy and Clinical Immunology 133 (6), 1564-1571, 2014 | 260 | 2014 |
A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice H Zhou, N Janghra, C Mitrpant, RL Dickinson, K Anthony, L Price, ... Human gene therapy 24 (3), 331-342, 2013 | 134 | 2013 |
Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy C Mitrpant, P Porensky, H Zhou, L Price, F Muntoni, S Fletcher, SD Wilton, ... PloS one 8 (4), e62114, 2013 | 95 | 2013 |
Structural variants may be a source of missing heritability in sALS F Theunissen, LL Flynn, RS Anderton, F Mastaglia, J Pytte, L Jiang, ... Frontiers in Neuroscience 14, 2020 | 53 | 2020 |
Complement-mediated muscle cell lysis: a possible mechanism of myonecrosis in anti-SRP associated necrotizing myopathy (ASANM) A Rojana-udomsart, C Mitrpant, C Bundell, L Price, YB Luo, V Fabian, ... Journal of neuroimmunology 264 (1-2), 65-70, 2013 | 49 | 2013 |
Novel STMN2 variant linked to amyotrophic lateral sclerosis risk and clinical phenotype F Theunissen, RS Anderton, FL Mastaglia, LL Flynn, SJ Winter, I James, ... Frontiers in aging neuroscience 13, 127, 2021 | 47 | 2021 |
p62 overexpression induces TDP-43 cytoplasmic mislocalisation, aggregation and cleavage and neuronal death AD Foster, LL Flynn, C Cluning, F Cheng, JM Davidson, A Lee, N Polain, ... Scientific reports 11 (1), 1-16, 2021 | 25 | 2021 |
Translational development of splice-modifying antisense oligomers S Fletcher, MI Bellgard, L Price, AP Akkari, SD Wilton Expert opinion on biological therapy 17 (1), 15-30, 2017 | 25 | 2017 |
Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript LL Flynn, C Mitrpant, IL Pitout, S Fletcher, SD Wilton Molecular Therapy-Nucleic Acids 11, 91-102, 2018 | 23 | 2018 |
Systematic approach to developing splice modulating antisense oligonucleotides MT Aung-Htut, CS McIntosh, KA Ham, IL Pitout, LL Flynn, K Greer, ... International journal of molecular sciences 20 (20), 5030, 2019 | 21 | 2019 |
Antisense-mediated splice intervention to treat human disease: the odyssey continues I Pitout, LL Flynn, SD Wilton, S Fletcher F1000Research 8, 2019 | 18 | 2019 |
Co-regulation of survival of motor neuron and Bcl-xL expression: implications for neuroprotection in spinal muscular atrophy RS Anderton, LL Price, BJ Turner, BP Meloni, C Mitrpant, FL Mastaglia, ... Neuroscience 220, 228-236, 2012 | 18 | 2012 |
Restoration of the CFTR function by splicing modulation B Kerem, MT Sinai, L Price, SD Wilton, S Fletcher US Patent 10,428,328, 2019 | 12 | 2019 |
Synucleinopathy in Amyotrophic Lateral Sclerosis: A Potential Avenue for Antisense Therapeutics? B Roberts, F Theunissen, FL Mastaglia, PA Akkari, LL Flynn International Journal of Molecular Sciences 23 (16), 9364, 2022 | 11 | 2022 |
Single stranded fully Modified-Phosphorothioate oligonucleotides can induce structured nuclear inclusions, alter nuclear protein localization and disturb the transcriptome In Vitro LL Flynn, R Li, IL Pitout, MT Aung-Htut, LM Larcher, JAL Cooper, KL Greer, ... Frontiers in genetics 13, Art. 791416, 2022 | 11 | 2022 |
Association of a structural variant within the SQSTM1 gene with amyotrophic lateral sclerosis J Pytte, RS Anderton, LL Flynn, F Theunissen, L Jiang, I Pitout, I James, ... Neurology Genetics 6 (2), 2020 | 11 | 2020 |
Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies LL Flynn, C Mitrpant, A Adams, IL Pitout, A Stirnweiss, S Fletcher, ... Biomedicines 9 (5), 552, 2021 | 10 | 2021 |