메뉴 건너뛰기




Volumn 867, Issue , 2012, Pages 143-167

Optimizing antisense oligonucleotides using phosphorodiamidate morpholino oligomers

Author keywords

Antisense oligonucleotides; DMD; Exon skipping; Functional analysis; Optimization; Phosphorodiamidate morpholino oligomers

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DNA; DYSTROPHIN; MORPHOLINO OLIGONUCLEOTIDE; RNA;

EID: 84859812797     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-61779-767-5_10     Document Type: Article
Times cited : (10)

References (28)
  • 1
    • 0023718118 scopus 로고
    • An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus
    • Monaco AP, Bertelson CJ, Liechti-Gallati S, Moser H, Kunkel LM (1988) An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics 2:90-95
    • (1988) Genomics , vol.2 , pp. 90-95
    • Monaco, A.P.1    Bertelson, C.J.2    Liechti-Gallati, S.3    Moser, H.4    Kunkel, L.M.5
  • 2
    • 38449095231 scopus 로고    scopus 로고
    • Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides
    • DOI 10.2741/2697
    • Bertoni C (2008) Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides. Front Biosci 13:517-527 (Pubitemid 351599762)
    • (2008) Frontiers in Bioscience , vol.13 , Issue.2 , pp. 517-527
    • Bertoni, C.1
  • 9
    • 34547116225 scopus 로고    scopus 로고
    • Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy
    • Aartsma-Rus A, Janson AA, van Ommen GJ, van Deutekom JCT (2007) Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy. BMC Med Genet 8: 43-51
    • (2007) BMC Med Genet , vol.8 , pp. 43-51
    • Aartsma-Rus, A.1    Janson, A.A.2    Van Ommen, G.J.3    Van Deutekom Jct4
  • 11
    • 13644267068 scopus 로고    scopus 로고
    • Towards a therapeutic inhibition of dystrophin exon 23 splicing in mdx mouse muscle induced by antisense oligoribonucleotides (splicomers): Target sequence optimisation using oligonucleotide arrays
    • DOI 10.1002/jgm.603
    • Graham IR, Hill VJ, Manoharan M, Inamati GB, Dickson G (2004) Towards a therapeutic inhibition of dystrophin exon 23 splicing in mdx mouse muscle induced by antisense oligoribonucleotides (splicomers): target sequence optimisation using oligonucleotide arrays. J Gene Med 6:1149-1158 (Pubitemid 40228127)
    • (2004) Journal of Gene Medicine , vol.6 , Issue.10 , pp. 1149-1158
    • Graham, I.R.1    Hill, V.J.2    Manoharan, M.3    Inamati, G.B.4    Dickson, G.5
  • 14
    • 50549093417 scopus 로고    scopus 로고
    • Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice
    • Jearawiriyapaisarn N, Moulton HM, Buckley B, Roberts J, Sazani P, Fucharoen S et al (2008) Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Mol Ther 16: 1624-1629
    • (2008) Mol Ther , vol.16 , pp. 1624-1629
    • Jearawiriyapaisarn, N.1    Moulton, H.M.2    Buckley, B.3    Roberts, J.4    Sazani, P.5    Fucharoen, S.6
  • 16
    • 69949107887 scopus 로고    scopus 로고
    • Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: A single-blind, placebocontrolled, dose-escalation, proof-of-concept study
    • Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C et al (2009) Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebocontrolled, dose-escalation, proof-of-concept study. Lancet Neurol 8(10):918-928
    • (2009) Lancet Neurol , vol.8 , Issue.10 , pp. 918-928
    • Kinali, M.1    Arechavala-Gomeza, V.2    Feng, L.3    Cirak, S.4    Hunt, D.5    Adkin, C.6
  • 17
    • 4844225667 scopus 로고    scopus 로고
    • Neutrally charged phosphorodiamidate Morpholino antisense oligomers: Uptake, efficacy and pharmacokinetics
    • DOI 10.2174/1389201043376706
    • Arora V, Devi GR, Iversen PL (2004) Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics. Curr Pharm Biotechnol 5:431-439 (Pubitemid 39317610)
    • (2004) Current Pharmaceutical Biotechnology , vol.5 , Issue.5 , pp. 431-439
    • Arora, V.1    Devi, G.R.2    Iversen, P.L.3
  • 19
    • 0043133425 scopus 로고    scopus 로고
    • Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle
    • DOI 10.1093/hmg/ddg196
    • Gebski BL, Mann CJ, Fletcher S, Wilton SD (2003) Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle. Hum Mol Genet 12:1801-1811 (Pubitemid 36944135)
    • (2003) Human Molecular Genetics , vol.12 , Issue.15 , pp. 1801-1811
    • Gebski, B.L.1    Mann, C.J.2    Fletcher, S.3    Wilton, S.D.4
  • 20
    • 32244443828 scopus 로고    scopus 로고
    • Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
    • DOI 10.1038/nm1345, PII NM1345
    • Alter J, Lou F, Rabinowitz A, Yin H, Rosenfeld J, Wilton SD et al (2006) Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 12:175-177 (Pubitemid 43214742)
    • (2006) Nature Medicine , vol.12 , Issue.2 , pp. 175-177
    • Alter, J.1    Lou, F.2    Rabinowitz, A.3    Yin, H.4    Rosenfeld, J.5    Wilton, S.D.6    Partridge, T.A.7    Qi, L.L.8
  • 21
    • 67650391305 scopus 로고    scopus 로고
    • Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice
    • Malerba A, Thorogood FC, Dickson G, Graham IR (2009) Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. Hum Gene Ther 20(9):955-965
    • (2009) Hum Gene Ther , vol.20 , Issue.9 , pp. 955-965
    • Malerba, A.1    Thorogood, F.C.2    Dickson, G.3    Graham, I.R.4
  • 23
    • 33745479703 scopus 로고    scopus 로고
    • Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
    • DOI 10.1038/sj.gt.3302800, PII 3302800
    • McClorey G, Moulton HM, Iversen PL, Fletcher S, Wilton SD (2006) Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther 13:1373-1381 (Pubitemid 44420632)
    • (2006) Gene Therapy , vol.13 , Issue.19 , pp. 1373-1381
    • McClorey, G.1    Moulton, H.M.2    Iversen, P.L.3    Fletcher, S.4    Wilton, S.D.5
  • 24
  • 25
    • 30844436415 scopus 로고    scopus 로고
    • Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: Indication for steric hindrance of SR protein binding sites
    • DOI 10.1089/oli.2005.15.284
    • Aartsma-Rus A, De Winter CL, Janson AAM, Kaman WE, van Ommen G-JB, Den Dunnen JT et al (2005) Functional analysis of 114 exoninternal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites. Oligonucleotides 15: 284-297 (Pubitemid 43108225)
    • (2005) Oligonucleotides , vol.15 , Issue.4 , pp. 284-297
    • Aartsma-Rus, A.1    De Winter, C.L.2    Janson, A.A.M.3    Kaman, W.E.4    Van Ommen, G.-J.B.5    Den Dunnen, J.T.6    Van Deutekom, J.C.T.7
  • 26
    • 34248511708 scopus 로고    scopus 로고
    • Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript
    • DOI 10.1038/sj.mt.6300095, PII 6300095
    • Wilton SD, Fall AM, Harding PL, McClorey G, Coleman C, Fletcher S (2007) Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript. Mol Ther 15:1288-1296 (Pubitemid 46965325)
    • (2007) Molecular Therapy , vol.15 , Issue.7 , pp. 1288-1296
    • Wilton, S.D.1    Fall, A.M.2    Harding, P.L.3    McClorey, G.4    Coleman, C.5    Fletcher, S.6
  • 27
  • 28
    • 61649109478 scopus 로고    scopus 로고
    • Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene
    • Popplewell LJ, Trollet C, Dickson G, Graham IR (2009) Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene. Mol Ther 17:554-561
    • (2009) Mol Ther , vol.17 , pp. 554-561
    • Popplewell, L.J.1    Trollet, C.2    Dickson, G.3    Graham, I.R.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.