Improving translational studies: lessons from rare neuromuscular diseases
Journal Article
Wells, D. J. Improving translational studies: lessons from rare neuromuscular diseases. Disease Models and Mechanisms, 8(10), 1175-7. https://doi.org/10.1242/dmm.022616
All Outputs (93)
THE TREAT-NMD ADVISORY COMMITTEE FOR THERAPEUTICS (TACT): AN INNOVATIVE DE-RISKING MODEL TO FOSTER ORPHAN DRUG DEVELOPMENT
Journal Article
Wells, D. J., Heslop, E., Csimma, C., Straub, V., McCall, J., Nagaraju, K., …Bushby, K. THE TREAT-NMD ADVISORY COMMITTEE FOR THERAPEUTICS (TACT): AN INNOVATIVE DE-RISKING MODEL TO FOSTER ORPHAN DRUG DEVELOPMENT. Neuromuscular Disorders, 25, S271-S271. https://doi.org/10.1016/j.nmd.2015.06.308
Translational and Regulatory Challenges for Exon Skipping Therapies
Journal Article
Aartsma-Rus, A., Ferlini, A., Goemans, N., Pasmooij, A. M. G., Wells, D. J., Bushby, K., …Balabanov, P. Translational and Regulatory Challenges for Exon Skipping Therapies. Human Gene Therapy, 25(10), 885-892. https://doi.org/10.1089/hum.2014.086
Report of the 2013 RSPCA/UFAW Rodent Welfare Group Meeting
Conference Proceeding
Hawkins, P., Littlefair, P., Gollege, H., Richardson, C., Allden, S., Boden, T., …Hubrecht, R. Report of the 2013 RSPCA/UFAW Rodent Welfare Group Meeting
Identification and validation of quantitative PCR reference genes suitable for normalising expression in normal and dystrophic cell culture models of myogenesis
Journal Article
Hildyard, J. C. W., & Wells, D. J. Identification and validation of quantitative PCR reference genes suitable for normalising expression in normal and dystrophic cell culture models of myogenesis. https://doi.org/10.1371/currents.md.faafdde4bea8df4aa7d06cd5553119a6
Mouse identification methods and potential welfare issues: a survey of current practice in the UK
Journal Article
Mazlan, N. H., Lopez-Salesansky, N., Burn, C. C., & Wells, D. J. Mouse identification methods and potential welfare issues: a survey of current practice in the UK
MUSCLE PHYSIOLOGY PROPERTIES OF MOUSE MODELS FOR DUCHENNE MUSCULAR DYSTROPHY
Journal Article
van Putten, M., Terry, R. L., Hulsker, M., Wells, K. E., Aartsma-Rus, A., & Wells, D. J. MUSCLE PHYSIOLOGY PROPERTIES OF MOUSE MODELS FOR DUCHENNE MUSCULAR DYSTROPHY. Neuromuscular Disorders, 24(9-10), 819-820. https://doi.org/10.1016/j.nmd.2014.06.098
ASSESSMENT OF NEUROMUSCULAR JUNCTION ABNORMALITIES INDUCED BY ALPHA-DYSTROGLYCAN GLYCOSYLATION DEFECTS
Journal Article
Fernandez-Fuente, M., Kim, J., Wells, D. J., & Brown, S. C. ASSESSMENT OF NEUROMUSCULAR JUNCTION ABNORMALITIES INDUCED BY ALPHA-DYSTROGLYCAN GLYCOSYLATION DEFECTS. Neuromuscular Disorders, 23(9-10), 781-782. https://doi.org/10.1016/j.nmd.2013.06.508
Response to the Comment published in ATLA, on the Declaration on Openness on Animal Research
Journal Article
Jarrett, W., Watts, G., Allan, C., Brazier, M., Clark, P., Deeny, A., …Steering Grp-Concordat Openness, . Response to the Comment published in ATLA, on the Declaration on Openness on Animal Research
PEPTIDE-PMO INDUCED EXON-SKIPPING RESTORES MUSCLE PHYSIOLOGY IN THE MDX MOUSE
Journal Article
Wells, K. E., Muses, S., Terry, R. L., Wood, M. J., Gait, M. J., & Wells, D. J. PEPTIDE-PMO INDUCED EXON-SKIPPING RESTORES MUSCLE PHYSIOLOGY IN THE MDX MOUSE. Neuromuscular Disorders, 23(9-10), 798-799. https://doi.org/10.1016/j.nmd.2013.06.562
MUSCLE PHYSIOLOGY PROPERTIES OF MOUSE MODELS FOR DUCHENNE MUSCULAR DYSTROPHY
Journal Article
van Putten, M., Terry, R., Hulsker, M., Wells, K. E., Aartsma-Rus, A., & Wells, D. J. MUSCLE PHYSIOLOGY PROPERTIES OF MOUSE MODELS FOR DUCHENNE MUSCULAR DYSTROPHY. Neuromuscular Disorders, 23(9-10), 840-840. https://doi.org/10.1016/j.nmd.2013.06.696
Preventing phosphorylation of dystroglycan ameliorates the dystrophic phenotype in mdx mouse
Journal Article
Miller, G., Moore, C. J., Terry, R. L., La Riviere, T., Mitchell, A., Piggott, R., …Winder, S. J. (in press). Preventing phosphorylation of dystroglycan ameliorates the dystrophic phenotype in mdx mouse. Human Molecular Genetics, 21(20), 4508-4520. https://doi.org/10.1093/hmg/dds293Loss of dystrophin protein due to mutations in the DMD gene causes Duchenne muscular dystrophy. Dystrophin loss also leads to the loss of the dystrophin glycoprotein complex (DGC) from the sarcolemma which contributes to the dystrophic phenotype. Tyr... Read More about Preventing phosphorylation of dystroglycan ameliorates the dystrophic phenotype in mdx mouse.
Effect of albumin and dextrose concentration on ultrasound and microbubble mediated gene transfection in vivo
Journal Article
Browning, R. J., Mulvana, H., Tang, M. X., Hajnal, J. V., Wells, D. J., & Eckersley, R. J. Effect of albumin and dextrose concentration on ultrasound and microbubble mediated gene transfection in vivo. Ultrasound in Medicine and Biology, 38(6), 1067-77. https://doi.org/10.1016/j.ultrasmedbio.2012.02.020