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Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment.
Betts CA, McClorey G, Healicon R, Hammond SM, Manzano R, Muses S, Ball V, Godfrey C, Merritt TM, van Westering T, O'Donovan L, Wells KE, Gait MJ, Wells DJ, Tyler D, Wood MJ. Betts CA, et al. Among authors: mcclorey g. Hum Mol Genet. 2019 Feb 1;28(3):396-406. doi: 10.1093/hmg/ddy346. Hum Mol Genet. 2019. PMID: 30281092 Free PMC article.
Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy.
Blain AM, Greally E, McClorey G, Manzano R, Betts CA, Godfrey C, O'Donovan L, Coursindel T, Gait MJ, Wood MJ, MacGowan GA, Straub VW. Blain AM, et al. Among authors: mcclorey g. PLoS One. 2018 Jun 18;13(6):e0198897. doi: 10.1371/journal.pone.0198897. eCollection 2018. PLoS One. 2018. PMID: 29912990 Free PMC article.
Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy.
Malerba A, Kang JK, McClorey G, Saleh AF, Popplewell L, Gait MJ, Wood MJ, Dickson G. Malerba A, et al. Among authors: mcclorey g. Mol Ther Nucleic Acids. 2012 Dec 18;1(12):e62. doi: 10.1038/mtna.2012.54. Mol Ther Nucleic Acids. 2012. PMID: 23250360 Free PMC article.
40 results