ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now?
Mejzini, Rita, Flynn, Loren L, Pitout, Ianthe L, Fletcher, Sue, Wilton, Steve D, Akkari, P Anthony
Published in Frontiers in neuroscience (06.12.2019)
Published in Frontiers in neuroscience (06.12.2019)
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Journal Article
Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping
Li, Dunhui, Mastaglia, Frank L., Fletcher, Sue, Wilton, Steve D.
Published in Trends in pharmacological sciences (Regular ed.) (01.11.2018)
Published in Trends in pharmacological sciences (Regular ed.) (01.11.2018)
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Journal Article
The FSHD atrophic myotube phenotype is caused by DUX4 expression
Vanderplanck, Céline, Ansseau, Eugénie, Charron, Sébastien, Stricwant, Nadia, Tassin, Alexandra, Laoudj-Chenivesse, Dalila, Wilton, Steve D, Coppée, Frédérique, Belayew, Alexandra
Published in PloS one (28.10.2011)
Published in PloS one (28.10.2011)
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Journal Article
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
Cirak, Sebahattin, MD, Arechavala-Gomeza, Virginia, PhD, Guglieri, Michela, MD, Feng, Lucy, PhD, Torelli, Silvia, PhD, Anthony, Karen, PhD, Abbs, Stephen, PhD, Garralda, Maria Elena, Prof, Bourke, John, MD, Wells, Dominic J, Prof, Dickson, George, Prof, Wood, Matthew JA, MD PhD, Wilton, Steve D, Prof, Straub, Volker, Prof, Kole, Ryszard, Prof, Shrewsbury, Stephen B, MD, Sewry, Caroline, Prof, Morgan, Jennifer E, PhD, Bushby, Kate, Prof, Muntoni, Francesco, Prof
Published in The Lancet (British edition) (13.08.2011)
Published in The Lancet (British edition) (13.08.2011)
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Journal Article
Functional improvement of dystrophic muscle by repression of utrophin: let-7c interaction
Mishra, Manoj K, Loro, Emanuele, Sengupta, Kasturi, Wilton, Steve D, Khurana, Tejvir S
Published in PloS one (18.10.2017)
Published in PloS one (18.10.2017)
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Journal Article
NEAT1 polyA-modulating antisense oligonucleotides reveal opposing functions for both long non-coding RNA isoforms in neuroblastoma
Naveed, Alina, Cooper, Jack A., Li, Ruohan, Hubbard, Alysia, Chen, Jingwei, Liu, Tao, Wilton, Steve D., Fletcher, Sue, Fox, Archa H.
Published in Cellular and molecular life sciences : CMLS (01.03.2021)
Published in Cellular and molecular life sciences : CMLS (01.03.2021)
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Journal Article
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Kinali, Maria, MD, Arechavala-Gomeza, Virginia, PhD, Feng, Lucy, PhD, Cirak, Sebahattin, MD, Hunt, David, FRCS, Adkin, Carl, PhD, Guglieri, Michela, MD, Ashton, Emma, PhD, Abbs, Stephen, PhD, Nihoyannopoulos, Petros, MD, Garralda, Maria Elena, MD, Rutherford, Mary, MD, Mcculley, Caroline, PhD, Popplewell, Linda, PhD, Graham, Ian R, PhD, Dickson, George, PhD, Wood, Matthew JA, MD, Wells, Dominic J, PhD, Wilton, Steve D, PhD, Kole, Ryszard, PhD, Straub, Volker, MD, Bushby, Kate, MD, Sewry, Caroline, PhD, Morgan, Jennifer E, PhD, Muntoni, Francesco, MD
Published in Lancet neurology (01.10.2009)
Published in Lancet neurology (01.10.2009)
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Journal Article
YAPping about and not forgetting TAZ
Callus, Bernard A., Finch‐Edmondson, Megan L., Fletcher, Sue, Wilton, Steve D.
Published in FEBS letters (01.02.2019)
Published in FEBS letters (01.02.2019)
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Journal Article
Antisense oligonucleotides and their applications in rare neurological diseases
McDowall, Simon, Aung-Htut, May, Wilton, Steve, Li, Dunhui
Published in Frontiers in neuroscience (23.09.2024)
Published in Frontiers in neuroscience (23.09.2024)
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Journal Article
Targeted Molecular Therapeutics for Parkinson's Disease: A Role for Antisense Oligonucleotides?
Li, Dunhui, Mastaglia, Frank L., Yau, Wai Yan, Chen, Shengdi, Wilton, Steve D., Akkari, Patrick A.
Published in Movement disorders (01.11.2022)
Published in Movement disorders (01.11.2022)
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Journal Article
Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52
Cale, Jessica M, Greer, Kane, Fletcher, Sue, Wilton, Steve D
Published in International journal of molecular sciences (27.03.2021)
Published in International journal of molecular sciences (27.03.2021)
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Journal Article
Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides
Aung-Htut, May T., Comerford, Iain, Johnsen, Russell, Foyle, Kerrie, Fletcher, Sue, Wilton, Steve D.
Published in Scientific reports (10.09.2019)
Published in Scientific reports (10.09.2019)
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A randomized ablation-based atrial fibrillation rhythm control versus rate control trial in patients with heart failure and high burden atrial fibrillation: The RAFT-AF trial rationale and design
Parkash, Ratika, Wells, George, Rouleau, Jean, Talajic, Mario, Essebag, Vidal, Skanes, Allan, Wilton, Steve B, Verma, Atul, Healey, Jeff S, Tang, Anthony SL
Published in The American heart journal (01.04.2021)
Published in The American heart journal (01.04.2021)
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Journal Article
Is Exon Skipping a Viable Therapeutic Approach for Vascular Ehlers-Danlos Syndrome with Mutations in COL3A1 Exon 10 or 15?
Utama, Sasiwimon, Cale, Jessica M, Mitrpant, Chalermchai, Fletcher, Sue, Wilton, Steve D, Aung-Htut, May T
Published in International journal of molecular sciences (13.08.2024)
Published in International journal of molecular sciences (13.08.2024)
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Journal Article
Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies
Toh, Zhi Yon Charles, Thandar Aung-Htut, May, Pinniger, Gavin, Adams, Abbie M, Krishnaswarmy, Sudarsan, Wong, Brenda L, Fletcher, Sue, Wilton, Steve D
Published in PloS one (08.01.2016)
Published in PloS one (08.01.2016)
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