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
All Outputs (4)
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/s10974-019-09535-9Duchenne 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/ddy346Duchenne 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.0211384The 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.