메뉴 건너뛰기




Volumn 1, Issue 1, 2011, Pages

Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy

Author keywords

[No Author keywords available]

Indexed keywords


EID: 80055038015     PISSN: None     EISSN: 20445040     Source Type: Journal    
DOI: 10.1186/2044-5040-1-8     Document Type: Review
Times cited : (23)

References (33)
  • 1
    • 0041582974 scopus 로고
    • Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide
    • 10.1073/pnas.75.1.280, 411230, 75545
    • Zamecnik PC, Stephenson ML. Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide. Proc Natl Acad Sci USA 1978, 75:280-284. 10.1073/pnas.75.1.280, 411230, 75545.
    • (1978) Proc Natl Acad Sci USA , vol.75 , pp. 280-284
    • Zamecnik, P.C.1    Stephenson, M.L.2
  • 2
    • 44649200454 scopus 로고    scopus 로고
    • Antisense, RNAi, and gene silencing strategies for therapy: mission possible or impossible?
    • 10.1016/j.drudis.2008.03.014, 2497463, 18549978
    • Rayburn ER, Zhang R. Antisense, RNAi, and gene silencing strategies for therapy: mission possible or impossible?. Drug Discov Today 2008, 13:513-521. 10.1016/j.drudis.2008.03.014, 2497463, 18549978.
    • (2008) Drug Discov Today , vol.13 , pp. 513-521
    • Rayburn, E.R.1    Zhang, R.2
  • 4
    • 0344420060 scopus 로고    scopus 로고
    • Dystrophin and mutations: one gene, several proteins, multiple phenotypes
    • 10.1016/S1474-4422(03)00585-4, 14636778
    • Muntoni F, Torelli S, Ferlini A. Dystrophin and mutations: one gene, several proteins, multiple phenotypes. Lancet Neurol 2003, 2:731-740. 10.1016/S1474-4422(03)00585-4, 14636778.
    • (2003) Lancet Neurol , vol.2 , pp. 731-740
    • Muntoni, F.1    Torelli, S.2    Ferlini, A.3
  • 5
    • 33749015734 scopus 로고    scopus 로고
    • Molecular mechanisms of muscular dystrophies: old and new players
    • 10.1038/nrm2024, 16971897
    • Davies KE, Nowak KJ. Molecular mechanisms of muscular dystrophies: old and new players. Nat Rev Mol Cell Biol 2006, 7:762-773. 10.1038/nrm2024, 16971897.
    • (2006) Nat Rev Mol Cell Biol , vol.7 , pp. 762-773
    • Davies, K.E.1    Nowak, K.J.2
  • 6
    • 0037160782 scopus 로고    scopus 로고
    • The muscular dystrophies
    • 10.1016/S0140-6736(02)07815-7, 11879882
    • Emery AE. The muscular dystrophies. Lancet 2002, 359:687-695. 10.1016/S0140-6736(02)07815-7, 11879882.
    • (2002) Lancet , vol.359 , pp. 687-695
    • Emery, A.E.1
  • 7
    • 0023718118 scopus 로고
    • An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus
    • 10.1016/0888-7543(88)90113-9, 3384440
    • Monaco AP, Bertelson CJ, Liechti-Gallati S, Moser H, Kunkel LM. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics 1988, 2:90-95. 10.1016/0888-7543(88)90113-9, 3384440.
    • (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
  • 8
    • 61649097962 scopus 로고    scopus 로고
    • Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
    • 10.1002/humu.20918, 19156838
    • Aartsma-Rus A, Fokkema I, Verschuuren J, Ginjaar I, van Deutekom J, van Ommen GJ, den Dunnen JT. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations. Hum Mutat 2009, 30:293-299. 10.1002/humu.20918, 19156838.
    • (2009) Hum Mutat , vol.30 , pp. 293-299
    • Aartsma-Rus, A.1    Fokkema, I.2    Verschuuren, J.3    Ginjaar, I.4    van Deutekom, J.5    van Ommen, G.J.6    den Dunnen, J.T.7
  • 9
    • 0030582315 scopus 로고    scopus 로고
    • Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence
    • 10.1006/bbrc.1996.1375, 8806654
    • Pramono ZA, Takeshim Y, Alimsardjono H, Ishii A, Takeda S, Matsuo M. Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence. Biochem Biophys Res Commun 1996, 226:445-449. 10.1006/bbrc.1996.1375, 8806654.
    • (1996) Biochem Biophys Res Commun , vol.226 , pp. 445-449
    • Pramono, Z.A.1    Takeshim, Y.2    Alimsardjono, H.3    Ishii, A.4    Takeda, S.5    Matsuo, M.6
  • 10
    • 0031800293 scopus 로고    scopus 로고
    • Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides
    • 10.1093/hmg/7.7.1083, 9618164
    • Dunckley MG, Manoharan M, Villiet P, Eperon IC, Dickson G. Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides. Hum Mol Genet 1998, 7:1083-1090. 10.1093/hmg/7.7.1083, 9618164.
    • (1998) Hum Mol Genet , vol.7 , pp. 1083-1090
    • Dunckley, M.G.1    Manoharan, M.2    Villiet, P.3    Eperon, I.C.4    Dickson, G.5
  • 12
    • 32844460899 scopus 로고    scopus 로고
    • Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide
    • 10.1002/jgm.838, 16285002
    • Fletcher S, Honeyman K, Fall AM, Harding PL, Johnsen RD, Wilton SD. Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide. J Gene Med 2006, 8:207-216. 10.1002/jgm.838, 16285002.
    • (2006) J Gene Med , vol.8 , pp. 207-216
    • Fletcher, S.1    Honeyman, K.2    Fall, A.M.3    Harding, P.L.4    Johnsen, R.D.5    Wilton, S.D.6
  • 14
    • 56649113066 scopus 로고    scopus 로고
    • Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle
    • 10.1093/nar/gkn671, 2582604, 18842625
    • Ivanova GD, Arzumanov A, Abes R, Yin H, Wood MJ, Lebleu B, Gait MJ. Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. Nucleic Acids Res 2008, 36:6418-6428. 10.1093/nar/gkn671, 2582604, 18842625.
    • (2008) Nucleic Acids Res , vol.36 , pp. 6418-6428
    • Ivanova, G.D.1    Arzumanov, A.2    Abes, R.3    Yin, H.4    Wood, M.J.5    Lebleu, B.6    Gait, M.J.7
  • 15
    • 37549022222 scopus 로고    scopus 로고
    • Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice
    • 10.1038/sj.mt.6300329, 17968354
    • Yin H, Lu Q, Wood M. Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice. Mol Ther 2008, 16:38-45. 10.1038/sj.mt.6300329, 17968354.
    • (2008) Mol Ther , vol.16 , pp. 38-45
    • Yin, H.1    Lu, Q.2    Wood, M.3
  • 16
    • 63449141811 scopus 로고    scopus 로고
    • Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs
    • 10.1002/ana.21627, 19288467
    • Yokota T, Lu QL, Partridge T, Kobayashi M, Nakamura A, Takeda S, Hoffman E. Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs. Ann Neurol 2009, 65:667-676. 10.1002/ana.21627, 19288467.
    • (2009) Ann Neurol , vol.65 , pp. 667-676
    • Yokota, T.1    Lu, Q.L.2    Partridge, T.3    Kobayashi, M.4    Nakamura, A.5    Takeda, S.6    Hoffman, E.7
  • 18
    • 0043133425 scopus 로고    scopus 로고
    • Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle
    • 10.1093/hmg/ddg196, 12874101
    • Gebski BL, Mann CJ, Fletcher S, Wilton SD. Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle. Hum Mol Genet 2003, 12:1801-1811. 10.1093/hmg/ddg196, 12874101.
    • (2003) Hum Mol Genet , vol.12 , pp. 1801-1811
    • Gebski, B.L.1    Mann, C.J.2    Fletcher, S.3    Wilton, S.D.4
  • 23
    • 79955808677 scopus 로고    scopus 로고
    • Current progress and preliminary results with the systemic administration trial of AVI-4658, a novel phosphorodiamidate morpholino oligomer (PMO) skipping dystrophin exon 51 in Duchenne muscular dystrophy (DMD)
    • Shrewsbury SB, Cirak S, Guglieri M, Bushby K, Muntoni F. Current progress and preliminary results with the systemic administration trial of AVI-4658, a novel phosphorodiamidate morpholino oligomer (PMO) skipping dystrophin exon 51 in Duchenne muscular dystrophy (DMD). Neuromuscul Disord 2010, 20:639-640.
    • (2010) Neuromuscul Disord , vol.20 , pp. 639-640
    • Shrewsbury, S.B.1    Cirak, S.2    Guglieri, M.3    Bushby, K.4    Muntoni, F.5
  • 24
    • 32244443828 scopus 로고    scopus 로고
    • Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
    • 10.1038/nm1345, 16444267
    • Alter J, Lou F, Rabinowitz A, Yin H, Rosenfeld J, Wilton SD, Partridge TA, Lu QL. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 2006, 12:175-177. 10.1038/nm1345, 16444267.
    • (2006) Nat Med , vol.12 , 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    Lu, Q.L.8
  • 26
    • 11844256373 scopus 로고    scopus 로고
    • Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
    • 10.1073/pnas.0406700102, 544058, 15608067
    • Lu QL, Rabinowitz A, Chen YC, Yokota T, Yin H, Alter J, Jadoon A, Bou-Gharios G, Partridge T. Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci USA 2005, 102:198-203. 10.1073/pnas.0406700102, 544058, 15608067.
    • (2005) Proc Natl Acad Sci USA , vol.102 , pp. 198-203
    • Lu, Q.L.1    Rabinowitz, A.2    Chen, Y.C.3    Yokota, T.4    Yin, H.5    Alter, J.6    Jadoon, A.7    Bou-Gharios, G.8    Partridge, T.9
  • 27
    • 74349109205 scopus 로고    scopus 로고
    • Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino
    • 10.1038/gt.2009.120, 19759562
    • Wu B, Lu P, Benrashid E, Malik S, Ashar J, Doran TJ, Lu QL. Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino. Gene Ther 17:132-140. 10.1038/gt.2009.120, 19759562.
    • Gene Ther , vol.17 , pp. 132-140
    • Wu, B.1    Lu, P.2    Benrashid, E.3    Malik, S.4    Ashar, J.5    Doran, T.J.6    Lu, Q.L.7
  • 28
    • 57049102809 scopus 로고    scopus 로고
    • Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function
    • 10.1093/hmg/ddn293, 18784278
    • Yin H, Moulton HM, Seow Y, Boyd C, Boutilier J, Iverson P, Wood MJ. Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. Hum Mol Genet 2008, 17:3909-3918. 10.1093/hmg/ddn293, 18784278.
    • (2008) Hum Mol Genet , vol.17 , pp. 3909-3918
    • Yin, H.1    Moulton, H.M.2    Seow, Y.3    Boyd, C.4    Boutilier, J.5    Iverson, P.6    Wood, M.J.7
  • 29
    • 74249109987 scopus 로고    scopus 로고
    • Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers
    • 10.1093/cvr/cvp335, 2802205, 19815563
    • Jearawiriyapaisarn N, Moulton HM, Sazani P, Kole R, Willis MS. Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers. Cardiovasc Res 2010, 85:444-453. 10.1093/cvr/cvp335, 2802205, 19815563.
    • (2010) Cardiovasc Res , vol.85 , pp. 444-453
    • Jearawiriyapaisarn, N.1    Moulton, H.M.2    Sazani, P.3    Kole, R.4    Willis, M.S.5
  • 31
    • 77958152873 scopus 로고    scopus 로고
    • Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy
    • 10.1016/j.bbamem.2010.02.012, 20170628
    • Moulton HM, Moulton JD. Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy. Biochim Biophys Acta 2010, 1798:2296-2303. 10.1016/j.bbamem.2010.02.012, 20170628.
    • (2010) Biochim Biophys Acta , vol.1798 , pp. 2296-2303
    • Moulton, H.M.1    Moulton, J.D.2
  • 32
    • 50549093417 scopus 로고    scopus 로고
    • Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice
    • 10.1038/mt.2008.120, 2671676, 18545222
    • Jearawiriyapaisarn N, Moulton HM, Buckley B, Roberts J, Sazani P, Fucharoen S, Iversen PL, Kole R. Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Mol Ther 2008, 16:1624-1629. 10.1038/mt.2008.120, 2671676, 18545222.
    • (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    Iversen, P.L.7    Kole, R.8
  • 33
    • 70350697818 scopus 로고    scopus 로고
    • A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice
    • 10.1093/hmg/ddp395, 19692354
    • Yin H, Moulton HM, Betts C, Seow Y, Boutilier J, Iverson PL, Wood MJ. A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice. Hum Mol Genet 2009, 18:4405-4414. 10.1093/hmg/ddp395, 19692354.
    • (2009) Hum Mol Genet , vol.18 , pp. 4405-4414
    • Yin, H.1    Moulton, H.M.2    Betts, C.3    Seow, Y.4    Boutilier, J.5    Iverson, P.L.6    Wood, M.J.7


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.