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., …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.