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Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.
Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P, Garralda ME, Rutherford M, McCulley C, Popplewell L, Graham IR, Dickson G, Wood MJ, Wells DJ, Wilton SD, Kole R, Straub V, Bushby K, Sewry C, Morgan JE, Muntoni F. Kinali M, et al. Among authors: adkin c. Lancet Neurol. 2009 Oct;8(10):918-28. doi: 10.1016/S1474-4422(09)70211-X. Epub 2009 Aug 25. Lancet Neurol. 2009. PMID: 19713152 Free PMC article. Clinical Trial.
Multiple exon skipping strategies to by-pass dystrophin mutations.
Adkin CF, Meloni PL, Fletcher S, Adams AM, Muntoni F, Wong B, Wilton SD. Adkin CF, et al. Neuromuscul Disord. 2012 Apr;22(4):297-305. doi: 10.1016/j.nmd.2011.10.007. Epub 2011 Dec 17. Neuromuscul Disord. 2012. PMID: 22182525 Free PMC article.
Targeted exon skipping to address "leaky" mutations in the dystrophin gene.
Fletcher S, Adkin CF, Meloni P, Wong B, Muntoni F, Kole R, Fragall C, Greer K, Johnsen R, Wilton SD. Fletcher S, et al. Among authors: adkin cf. Mol Ther Nucleic Acids. 2012 Oct 16;1(10):e48. doi: 10.1038/mtna.2012.40. Mol Ther Nucleic Acids. 2012. PMID: 23344648 Free PMC article.