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Page 1
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, Dickson G, Wood MJ, Wilton SD, Straub V, Kole R, Shrewsbury SB, Sewry C, Morgan JE, Bushby K, Muntoni F. Cirak S, et al. Among authors: kole r. Lancet. 2011 Aug 13;378(9791):595-605. doi: 10.1016/S0140-6736(11)60756-3. Epub 2011 Jul 23. Lancet. 2011. PMID: 21784508 Free PMC article. Clinical Trial.
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: kole r. 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.
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: kole r. 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.
Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.
Echigoya Y, Nakamura A, Nagata T, Urasawa N, Lim KRQ, Trieu N, Panesar D, Kuraoka M, Moulton HM, Saito T, Aoki Y, Iversen P, Sazani P, Kole R, Maruyama R, Partridge T, Takeda S, Yokota T. Echigoya Y, et al. Among authors: kole r. Proc Natl Acad Sci U S A. 2017 Apr 18;114(16):4213-4218. doi: 10.1073/pnas.1613203114. Epub 2017 Apr 3. Proc Natl Acad Sci U S A. 2017. PMID: 28373570 Free PMC article.
141 results