메뉴 건너뛰기




Volumn 19, Issue 7, 2011, Pages 1295-1303

Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MORPHOLINE DERIVATIVE; PEPTIDE NUCLEIC ACID; PHOSPHORODIAMIDATE MORPHOLINO; UNCLASSIFIED DRUG;

EID: 79959995255     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.79     Document Type: Article
Times cited : (112)

References (32)
  • 1
    • 77950793623 scopus 로고    scopus 로고
    • RNA-targeted splice-correction therapy for neuromuscular disease
    • Wood, MJ, Gait, MJ and Yin, H (2010). RNA-targeted splice-correction therapy for neuromuscular disease. Brain 133(Pt 4): 957-972.
    • (2010) Brain , vol.133 , Issue.PART 4 , pp. 957-972
    • Wood, M.J.1    Gait, M.J.2    Yin, H.3
  • 2
    • 77953017904 scopus 로고    scopus 로고
    • Toward an oligonucleotide therapy for Duchenne muscular dystrophy: A complex development challenge
    • Wood, MJ (2010). Toward an oligonucleotide therapy for Duchenne muscular dystrophy: a complex development challenge. Sci Transl Med 2: 25ps15.
    • (2010) Sci Transl Med , vol.2
    • Wood, M.J.1
  • 6
    • 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
  • 7
    • 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 and 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
  • 11
    • 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 and 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
  • 12
    • 37549022222 scopus 로고    scopus 로고
    • Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice
    • Yin, H, Lu, Q and Wood, M (2008). Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice. Mol Ther 16: 38-45.
    • (2008) Mol Ther , vol.16 , pp. 38-45
    • Yin, H.1    Lu, Q.2    Wood, M.3
  • 13
    • 57049102809 scopus 로고    scopus 로고
    • Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function
    • DOI 10.1093/hmg/ddn293
    • Yin, H, Moulton, HM, Seow, Y, Boyd, C, Boutilier, J, Iverson, P et al. (2008). Cellpenetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. Hum Mol Genet 17: 3909-3918. (Pubitemid 352762853)
    • (2008) Human Molecular Genetics , vol.17 , Issue.24 , 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.A.7
  • 14
    • 70350697818 scopus 로고    scopus 로고
    • A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice
    • Yin, H, Moulton, HM, Betts, C, Seow, Y, Boutilier, J, Iverson, PL et al. (2009). A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice. Hum Mol Genet 18: 4405-4414.
    • (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
  • 15
    • 77950898446 scopus 로고    scopus 로고
    • In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy
    • Wang, Q, Yin, H, Camelliti, P, Betts, C, Moulton, H, Lee, H et al. (2010). In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy. J Gene Med 12: 354-364.
    • (2010) J Gene Med , vol.12 , pp. 354-364
    • Wang, Q.1    Yin, H.2    Camelliti, P.3    Betts, C.4    Moulton, H.5    Lee, H.6
  • 18
    • 69949107887 scopus 로고    scopus 로고
    • 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 et al. (2009). 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. Lancet Neurol 8: 918-928.
    • (2009) Lancet Neurol , vol.8 , pp. 918-928
    • Kinali, M.1    Arechavala-Gomeza, V.2    Feng, L.3    Cirak, S.4    Hunt, D.5    Adkin, C.6
  • 19
    • 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
  • 20
    • 54449095504 scopus 로고    scopus 로고
    • Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
    • Wu, B, Moulton, HM, Iversen, PL, Jiang, J, Li, J, Li, J et al. (2008). Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer. Proc Natl Acad Sci USA 105: 14814-14819.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 14814-14819
    • Wu, B.1    Moulton, H.M.2    Iversen, P.L.3    Jiang, J.4    Li, J.5    Li, J.6
  • 21
    • 77957605909 scopus 로고    scopus 로고
    • Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO
    • Yin, H, Moulton, HM, Betts, C, Merritt, T, Seow, Y, Ashraf, S et al. (2010). Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO. Mol Ther 18: 1822-1829.
    • (2010) Mol Ther , vol.18 , pp. 1822-1829
    • Yin, H.1    Moulton, H.M.2    Betts, C.3    Merritt, T.4    Seow, Y.5    Ashraf, S.6
  • 22
    • 56649113066 scopus 로고    scopus 로고
    • Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle
    • Ivanova, GD, Arzumanov, A, Abes, R, Yin, H, Wood, MJ, Lebleu, B et al. (2008). Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. Nucleic Acids Res 36: 6418-6428.
    • (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
  • 23
    • 77950518145 scopus 로고    scopus 로고
    • Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse
    • Yin, H, Betts, C, Saleh, AF, Ivanova, GD, Lee, H, Seow, Y et al. (2010). Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse. Mol Ther 18: 819-827.
    • (2010) Mol Ther , vol.18 , pp. 819-827
    • Yin, H.1    Betts, C.2    Saleh, A.F.3    Ivanova, G.D.4    Lee, H.5    Seow, Y.6
  • 25
    • 0034687238 scopus 로고    scopus 로고
    • Alternative splicing of dystrobrevin regulates the stoichiometry of syntrophin binding to the dystrophin protein complex
    • Newey, SE, Benson, MA, Ponting, CP, Davies, KE and Blake, DJ (2000). Alternative splicing of dystrobrevin regulates the stoichiometry of syntrophin binding to the dystrophin protein complex. Curr Biol 10: 1295-1298.
    • (2000) Curr Biol , vol.10 , pp. 1295-1298
    • Newey, S.E.1    Benson, M.A.2    Ponting, C.P.3    Davies, K.E.4    Blake, D.J.5
  • 26
    • 0023780812 scopus 로고
    • Serum CK, calcium, magnesium, and oxidative phosphorylation in mdx mouse muscular dystrophy
    • Glesby, MJ, Rosenmann, E, Nylen, EG and Wrogemann, K (1988). Serum CK, calcium, magnesium, and oxidative phosphorylation in mdx mouse muscular dystrophy. Muscle Nerve 11: 852-856.
    • (1988) Muscle Nerve , vol.11 , pp. 852-856
    • Glesby, M.J.1    Rosenmann, E.2    Nylen, E.G.3    Wrogemann, K.4
  • 27
    • 77950658740 scopus 로고    scopus 로고
    • Heart-specific overexpression of CUGBP1 reproduces functional and molecular abnormalities of myotonic dystrophy type 1
    • Koshelev, M, Sarma, S, Price, RE, Wehrens, XH and Cooper, TA (2010). Heart-specific overexpression of CUGBP1 reproduces functional and molecular abnormalities of myotonic dystrophy type 1. Hum Mol Genet 19: 1066-1075.
    • (2010) Hum Mol Genet , vol.19 , pp. 1066-1075
    • Koshelev, M.1    Sarma, S.2    Price, R.E.3    Wehrens, X.H.4    Cooper, T.A.5
  • 28
    • 72849117063 scopus 로고    scopus 로고
    • PKC inhibition ameliorates the cardiac phenotype in a mouse model of myotonic dystrophy type 1
    • Wang, GS, Kuyumcu-Martinez, MN, Sarma, S, Mathur, N, Wehrens, XH and Cooper, TA (2009). PKC inhibition ameliorates the cardiac phenotype in a mouse model of myotonic dystrophy type 1. J Clin Invest 119: 3797-3806.
    • (2009) J Clin Invest , vol.119 , pp. 3797-3806
    • Wang, G.S.1    Kuyumcu-Martinez, M.N.2    Sarma, S.3    Mathur, N.4    Wehrens, X.H.5    Cooper, T.A.6
  • 29
    • 74349109205 scopus 로고    scopus 로고
    • Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino
    • Wu, B, Lu, P, Benrashid, E, Malik, S, Ashar, J, Doran, TJ et al. (2010). Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino. Gene Ther 17: 132-140.
    • (2010) 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
  • 30
    • 67349230291 scopus 로고    scopus 로고
    • Microbubble stability is a major determinant of the efficiency of ultrasound and microbubble mediated in vivo gene transfer
    • Alter, J, Sennoga, CA, Lopes, DM, Eckersley, RJ and Wells, DJ (2009). Microbubble stability is a major determinant of the efficiency of ultrasound and microbubble mediated in vivo gene transfer. Ultrasound Med Biol 35: 976-984.
    • (2009) Ultrasound Med Biol , vol.35 , pp. 976-984
    • Alter, J.1    Sennoga, C.A.2    Lopes, D.M.3    Eckersley, R.J.4    Wells, D.J.5
  • 31
    • 0023790515 scopus 로고
    • A rat antibody against a structure functionally related to the mouse T-cell receptor/T3 complex
    • DOI 10.1007/BF00355379
    • Tomonari, K (1988). A rat antibody against a structure functionally related to the mouse T-cell receptor/T3 complex. Immunogenetics 28: 455-458. (Pubitemid 18262880)
    • (1988) Immunogenetics , vol.28 , Issue.6 , pp. 455-458
    • Tomonari, K.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.