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All Outputs (57)

Animal Research beyond the Laboratory: Report from a Workshop on Places Other than Licensed Establishments (POLEs) in the UK (2020)
Journal Article
Palmer, A., Greenhough, B., Hobson-West, P., Message, R., Aegerter, J. N., Belshaw, Z., Dennison, N., Dickey, R., Lane, J., Lorimer, J., Millar, K., Newman, C., Pullen, K., Reynolds, S. J., Wells, D. J., Witt, M. J., & Wolfensohn, S. (2020). Animal Research beyond the Laboratory: Report from a Workshop on Places Other than Licensed Establishments (POLEs) in the UK. Animals, 10(10), e1868

Research involving animals that occurs outside the laboratory raises an array of unique challenges. With regard to UK legislation, however, it receives only limited attention in terms of official guidelines, support, and statistics, which are unsurpr... Read More about Animal Research beyond the Laboratory: Report from a Workshop on Places Other than Licensed Establishments (POLEs) in the UK.

Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT) (2020)
Journal Article
Willmann, R., Lee, J., Turner, C., Nagaraju, K., Aartsma-Rus, A., Wells, D. J., Wagner, K. R., Csimma, C., Straub, V., Grounds, M. D., & De Luca, A. (2020). Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT). Disease Models and Mechanisms, 13(2), https://doi.org/10.1242/dmm.042903

Clinical trials for rare neuromuscular diseases imply, among other investments, a high emotional burden for the whole disease community. Translation of data from preclinical studies to justify any clinical trial must be carefully pondered in order to... Read More about Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT).

A decade of optimizing drug development for rare neuromuscular disorders through TACT (2019)
Journal Article
Wagner, K. R., De Luca, A., Caizergues, D., Dowling, J., Goemans, N., Gordish-Dressman, H., Grounds, M. D., Kelly, M., Mayhew, A., McNally, E. M., Zoetis, T., Lee, J., Turner, C., Wells, D. J., Csimma, C., & Straub, V. (2019). A decade of optimizing drug development for rare neuromuscular disorders through TACT. Nature Reviews Drug Discovery, 19(1-2), https://doi.org/10.1038/d41573-019-00199-1

What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy? (2019)
Journal Article
Wells, D. J. (2019). What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy?. Journal of Muscle Research and Cell Motility, 40(2), 141-150. https://doi.org/10.1007/s10974-019-09535-9

Duchenne muscular dystrophy (DMD) is a fatal X-linked muscle wasting disease. The disease is due to mutations in the DMD gene that encodes for a large intracellular protein called dystrophin. Dystrophin plays a critical role in linking the internal c... Read More about What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy?.

Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment (2019)
Journal Article
Betts, C. A., McClorey, G., Healicon, R., Hammond, S. M., Manzano, R., Muses, S., Ball, V., Godfrey, C., Merritt, T. M., Westering, T., O'Donovan, L., Wells, K. E., Gait, M. J., Wells, D. J., Tyler, D., & Wood, M. J. (2019). Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment. Human Molecular Genetics, 28(3), 396-406. https://doi.org/10.1093/hmg/ddy346

Duchenne muscular dystrophy (DMD) is caused by loss of dystrophin protein, leading to progressive muscle weakness and premature death due to respiratory and/or cardiac complications. Cardiac involvement is characterized by progressive dilated cardiom... Read More about Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment.

Identification of qPCR reference genes suitable for normalizing gene expression in the mdx mouse model of Duchenne muscular dystrophy (2019)
Journal Article
Hildyard, J. C. W., Finch, A. M., & Wells, D. J. (2019). Identification of qPCR reference genes suitable for normalizing gene expression in the mdx mouse model of Duchenne muscular dystrophy. PLoS ONE, 14(1), e0211384. https://doi.org/10.1371/journal.pone.0211384

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 muscles of this animal plays a key role in the study of pathogenesis and disease progression... Read More about Identification of qPCR reference genes suitable for normalizing gene expression in the mdx mouse model of Duchenne muscular dystrophy.

"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic (2018)
Journal Article
Gordish-Dressman, H., Willmann, R., Dalle Pazze, L., Kreibich, A., van Putten, M., Heydemann, A., Bogdanik, L., Davies, K., Demonbruen, A., Duan, D., Elsey, D., Fukada, S., Girgenrath, S., Gonzalez, P., Grounds, M., Nichols, A., Partridge, T., Passini, M., Sanarica, F., Schnell, F., …Aartsma-Rus, A. (2018). "Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic. Journal of Neuromuscular Diseases, 5(4), 407-414. https://doi.org/10.3233/JND-180324

A new line of dystrophic mdx mice on the DBA/2J (D2) background has emerged as a candidate to study the efficacy of therapeutic approaches for Duchenne muscular dystrophy (DMD). These mice harbor genetic polymorphisms that appear to increase the seve... Read More about "Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic.

Focus on the Role of D-serine and D-amino Acid Oxidase in Amyotrophic Lateral Sclerosis/Motor Neuron Disease (ALS) (2018)
Journal Article
Kondori, N. R., Paul, P., Robbins, J. P., Liu, K., Hildyard, J. C. W., Wells, D. J., & De Belleroche, J. S. (2018). Focus on the Role of D-serine and D-amino Acid Oxidase in Amyotrophic Lateral Sclerosis/Motor Neuron Disease (ALS). Frontiers in Molecular Biosciences, 5(8), https://doi.org/10.3389/fmolb.2018.00008

Highly pathogenic avian influenza (HPAI) H5N1 virus has been circulating in Vietnam since 2003, while outbreaks of HPAI H5N6 virus are more recent, having only been reported since 2014. Although the spatial distribution of H5N1 outbreaks and risk fac... Read More about Focus on the Role of D-serine and D-amino Acid Oxidase in Amyotrophic Lateral Sclerosis/Motor Neuron Disease (ALS).

Growth differentiation factor-15 is associated with muscle mass in chronic obstructive pulmonary disease and promotes muscle wasting in vivo (2016)
Journal Article
Patel, M. S., Lee, J., Baz, M., Wells, C. E., Bloch, S., Lewis, A., Donaldson, A. V., Garfield, B. E., Hopkinson, N. S., Natanek, A., Man, W. D. C., Wells, D. J., Baker, E. H., Polkey, M. I., & Kemp, P. R. (2016). Growth differentiation factor-15 is associated with muscle mass in chronic obstructive pulmonary disease and promotes muscle wasting in vivo. Journal of Cachexia, Sarcopenia and Muscle, 7(4), 436-448. https://doi.org/10.1002/jcsm.12096

Transgenic Rescue of the LARGEmyd Mouse: A LARGE Therapeutic Window? (2016)
Journal Article
Hildyard, J. C. W., Lacey, E., Booler, H., Hopkinson, M., Wells, D. J., & Brown, S. C. (2016). Transgenic Rescue of the LARGEmyd Mouse: A LARGE Therapeutic Window?. PLoS ONE, 11(7), e0159853. https://doi.org/10.1371/journal.pone.0159853

LARGE is a glycosyltransferase involved in glycosylation of α-dystroglycan (α-DG). Absence of this protein in the LARGEmyd mouse results in α-DG hypoglycosylation, and is associated with central nervous system abnormalities and progressive muscular d... Read More about Transgenic Rescue of the LARGEmyd Mouse: A LARGE Therapeutic Window?.

Musculoskeletal Geometry, Muscle Architecture and Functional Specialisations of the Mouse Hindlimb (2016)
Journal Article
Charles, J. P., Cappellari, O., Spence, A. J., Hutchinson, J. R., & Wells, D. J. (2016). Musculoskeletal Geometry, Muscle Architecture and Functional Specialisations of the Mouse Hindlimb. PLoS ONE, 11(4), https://doi.org/10.1371/journal.pone.0147669

Mice are one of the most commonly used laboratory animals, with an extensive array of disease models in existence, including for many neuromuscular diseases. The hindlimb is of particular interest due to several close muscle analogues/homologues to h... Read More about Musculoskeletal Geometry, Muscle Architecture and Functional Specialisations of the Mouse Hindlimb.

FHL1 activates myostatin signalling in skeletal muscle and promotes atrophy (2015)
Journal Article
Lee, J. Y., Lori, D., Wells, D. J., & Kemp, P. R. (2015). FHL1 activates myostatin signalling in skeletal muscle and promotes atrophy. FEBS Open Bio, 5, 753-762. https://doi.org/10.1016/j.fob.2015.08.011

Myostatin is a TGFβ family ligand that reduces muscle mass. In cancer cells, TGFβ signalling is increased by the protein FHL1. Consequently, FHL1 may promote signalling by myostatin. We therefore tested the ability of FHL1 to regulate myostatin funct... Read More about FHL1 activates myostatin signalling in skeletal muscle and promotes atrophy.

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse (2015)
Journal Article
Godfrey, C., Muses, S., McClorey, G., Wells, K. E., Coursindel, T., Terry, R. L., Betts, C., Hammond, S., O'Donovan, L., Hildyard, J. C. W., El Andaloussi, S., Gait, M. J., Wood, M. J., & Wells, D. J. (2015). How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse. Human Molecular Genetics, 24(15), 4225-4237. https://doi.org/10.1093/hmg/ddv155

Measuring Clinical Effectiveness of Medicinal Products for the Treatment of Duchenne Muscular Dystrophy (2015)
Journal Article
Lynn, S., Aartsma-Rus, A., Furlong, P., Goemans, N., De Luca, A., Mayhew, A., McDonald, C., Mercuri, E., Muntoni, F., Pohlschmidt, M., Verschuuren, J., Voit, T., Vroom, E., Wells, D. J., & Straub, V. (2015). Measuring Clinical Effectiveness of Medicinal Products for the Treatment of Duchenne Muscular Dystrophy. Neuromuscular Disorders, 25(1), 96-105. https://doi.org/10.1016/j.nmd.2014.09.003

Poloxomer 188 Has a Deleterious Effect on Dystrophic Skeletal Muscle Function (2014)
Journal Article
Terry, R. L., Kaneb, H. M., & Wells, D. J. (2014). Poloxomer 188 Has a Deleterious Effect on Dystrophic Skeletal Muscle Function. PLoS ONE, 9(3), e91221. https://doi.org/10.1371/journal.pone.0091221

Duchenne muscular dystrophy (DMD) is an X-linked, fatal muscle wasting disease for which there is currently no cure and limited palliative treatments. Poloxomer 188 (P188) is a tri-block copolymer that has been proposed as a potential treatment for c... Read More about Poloxomer 188 Has a Deleterious Effect on Dystrophic Skeletal Muscle Function.

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-esclation, proof-of-concept study (2009)
Journal Article
Kinali, M., Arechavala-Gomeza, V., Feng, L., Cirak, S., Hunt, D., Adkin, C., Guglieri, M., Ashton, E., Abbs, S., Nihoyannopoulos, P., Garralda, M. E., Rutherford, M., McCulley, C., Popplewell, L., Graham, I. R., Dickson, G., Wood, M. J., Wells, D. J., Wilton, S. D., Kole, R., …Muntoni, F. (2009). Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-esclation, proof-of-concept study. https://doi.org/10.1016/S1474-4422%2809%2970211-X