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., Vroom, E., & 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
All Outputs (65)
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. Animal Technology and Welfare, 13(1), 1-10
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., Lee, N., Moon, L., Robinson, V., Taylor, D., Wells, D., Willis, L. P., & Steering Grp-Concordat Openness. Response to the Comment published in ATLA, on the Declaration on Openness on Animal Research
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., Dear, T. N., Wells, D. J., & 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.