Search Results - "Wells, Dominic J"

Tracking progress: an update on animal models for Duchenne muscular dystrophy by Wells, Dominic J

“…Duchenne muscular dystrophy (DMD) is a progressive, fatal, X-linked monogenic muscle disorder caused by mutations in the gene. In order to test treatments for…”
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Identification of qPCR reference genes suitable for normalizing gene expression in the mdx mouse model of Duchenne muscular dystrophy by Hildyard, John C W, Finch, Amber M, Wells, Dominic J

“…The mdx mouse is the most widely-used animal model of the human disease Duchenne muscular dystrophy, and quantitative PCR analysis of gene expression in the…”
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Three-Dimensional Human iPSC-Derived Artificial Skeletal Muscles Model Muscular Dystrophies and Enable Multilineage Tissue Engineering by Maffioletti, Sara Martina, Sarcar, Shilpita, Henderson, Alexander B.H., Mannhardt, Ingra, Pinton, Luca, Moyle, Louise Anne, Steele-Stallard, Heather, Cappellari, Ornella, Wells, Kim E., Ferrari, Giulia, Mitchell, Jamie S., Tyzack, Giulia E., Kotiadis, Vassilios N., Khedr, Moustafa, Ragazzi, Martina, Wang, Weixin, Duchen, Michael R., Patani, Rickie, Zammit, Peter S., Wells, Dominic J., Eschenhagen, Thomas, Tedesco, Francesco Saverio

“…Generating human skeletal muscle models is instrumental for investigating muscle pathology and therapy. Here, we report the generation of three-dimensional…”
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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 by 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

“…Summary Background We report clinical safety and biochemical efficacy from a dose-ranging study of intravenously administered AVI-4658 phosphorodiamidate…”
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Musculoskeletal Geometry, Muscle Architecture and Functional Specialisations of the Mouse Hindlimb by Charles, James P, Cappellari, Ornella, Spence, Andrew J, Hutchinson, John R, Wells, Dominic J

“…Mice are one of the most commonly used laboratory animals, with an extensive array of disease models in existence, including for many neuromuscular diseases…”
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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 by 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

“…Summary Background Mutations that disrupt the open reading frame and prevent full translation of DMD , the gene that encodes dystrophin, underlie the fatal…”
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Multiplex in situ hybridization within a single transcript: RNAscope reveals dystrophin mRNA dynamics by Hildyard, John C W, Rawson, Faye, Wells, Dominic J, Piercy, Richard J

“…Dystrophin plays a vital role in maintaining muscle health, yet low mRNA expression, lengthy transcription time and the limitations of traditional in-situ…”
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Electroporation and ultrasound enhanced non-viral gene delivery in vitro and in vivo by Wells, Dominic J

“…Non-viral vectors are less efficient than the use of viral vectors for delivery of genetic material to cells in vitro and especially in vivo. However, viral…”
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Serum inflammatory cytokines as disease biomarkers in the DE50-MD dog model of Duchenne muscular dystrophy by Riddell, Dominique O, Hildyard, John C W, Harron, Rachel C M, Hornby, Natasha L, Wells, Dominic J, Piercy, Richard J

“…Duchenne muscular dystrophy (DMD) is a fatal muscle-wasting disease, caused by mutations in the dystrophin gene, characterised by cycles of muscle…”
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Metformin treatment has no beneficial effect in a dose-response survival study in the SOD1(G93A) mouse model of ALS and is harmful in female mice by Kaneb, Hannah M, Sharp, Paul S, Rahmani-Kondori, Nazanin, Wells, Dominic J

“…Amyotrophic Lateral Sclerosis (ALS) is a devastating neurological disorder characterized by selective degeneration of upper and lower motor neurons. The…”
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Longitudinal assessment of skeletal muscle functional mechanics in the DE50-MD dog model of Duchenne muscular dystrophy by Riddell, Dominique O, Hildyard, John C W, Harron, Rachel C M, Taylor-Brown, Frances, Kornegay, Joe N, Wells, Dominic J, Piercy, Richard J

“…Duchenne muscular dystrophy (DMD), caused by mutations in the dystrophin (DMD) gene, is associated with fatal muscle degeneration and atrophy. Patients with…”
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Validation of DE50-MD dogs as a model for the brain phenotype of Duchenne muscular dystrophy by Crawford, Abbe H, Hildyard, John C W, Rushing, Sophie A M, Wells, Dominic J, Diez-Leon, Maria, Piercy, Richard J

“…Duchenne muscular dystrophy (DMD), a fatal musculoskeletal disease, is associated with neurodevelopmental disorders and cognitive impairment caused by brain…”
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Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT) by Willmann, Raffaella, Lee, Joanne, Turner, Cathy, Nagaraju, Kanneboyina, Aartsma-Rus, Annemieke, Wells, Dominic J, Wagner, Kathryn R, Csimma, Cristina, Straub, Volker, Grounds, Miranda D, De Luca, Annamaria

“…Clinical trials for rare neuromuscular diseases imply, among other investments, a high emotional burden for the whole disease community. Translation of data…”
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Characterisation of the pathogenic effects of the in vivo expression of an ALS-linked mutation in D-amino acid oxidase: Phenotype and loss of spinal cord motor neurons by Kondori, Nazanin Rahmani, Paul, Praveen, Robbins, Jacqueline P, Liu, Ke, Hildyard, John C W, Wells, Dominic J, de Belleroche, Jacqueline S

“…Amyotrophic lateral sclerosis (ALS) is the most common adult-onset neuromuscular disorder characterised by selective loss of motor neurons leading to fatal…”
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Extracellular Release of the Atheroprotective Heat Shock Protein 27 Is Mediated by Estrogen and Competitively Inhibits acLDL Binding to Scavenger Receptor-A by Rayner, Katey, Chen, Yong-Xiang, McNulty, Melissa, Simard, Trevor, Zhao, Xioaling, Wells, Dominic J, de Belleroche, Jacqueline, O’Brien, Edward R

“…We recently identified heat shock protein 27 (HSP27) as an estrogen receptor beta (ERβ)-associated protein and noted its role as a biomarker for…”
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Identification and validation of quantitative PCR reference genes suitable for normalizing expression in normal and dystrophic cell culture models of myogenesis by Hildyard, John C W, Wells, Dominic J

“…The coordinated differentiation of myoblasts to mature muscle is essential for muscle development and repair, and study of the myogenic program in health and…”
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A new extensively characterised conditionally immortal muscle cell-line for investigating therapeutic strategies in muscular dystrophies by Muses, Sofia, Morgan, Jennifer E, Wells, Dominic J

“…A new conditionally immortal satellite cell-derived cell-line, H2K 2B4, was generated from the H2K(b)-tsA58 immortomouse. Under permissive conditions H2K 2B4…”
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The Pen Is Milder Than the Blade: Identification Marking Mice Using Ink on the Tail Appears More Humane Than Ear-Punching Even with Local Anaesthetic by Burn, Charlotte C., Mazlan, Nur H. B., Chancellor, Natalie, Wells, Dominic J.

“…Identification marking mice commonly involves ear-punching with or without anaesthetic, or tail-marking with ink. To identify which is most humane, we marked…”
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The transgenic expression of LARGE exacerbates the muscle phenotype of dystroglycanopathy mice by Whitmore, Charlotte, Fernandez-Fuente, Marta, Booler, Helen, Parr, Callum, Kavishwar, Manoli, Ashraf, Attia, Lacey, Erica, Kim, Jihee, Terry, Rebecca, Ackroyd, Mark R, Wells, Kim E, Muntoni, Francesco, Wells, Dominic J, Brown, Susan C

“…Mutations in fukutin-related protein (FKRP) underlie a group of muscular dystrophies associated with the hypoglycosylation of α-dystroglycan (α-DG), a…”
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Codon and mRNA Sequence Optimization of Microdystrophin Transgenes Improves Expression and Physiological Outcome in Dystrophic mdx Mice Following AAV2/8 Gene Transfer by Foster, Helen, Sharp, Paul S, Athanasopoulos, Takis, Trollet, Capucine, Graham, Ian R, Foster, Keith, Wells, Dominic J, Dickson, George

“…Duchenne muscular dystrophy is a fatal muscle-wasting disorder. Lack of dystrophin compromises the integrity of the sarcolemma and results in myofibers that…”
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