메뉴 건너뛰기




Volumn 20, Issue 6, 2011, Pages 1293-1304

Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA

Author keywords

Animal model; For gene therapy; Neurodegenerative disease; Spinal Muscular Atrophy (SMA); Transgenic pigs

Indexed keywords

SINGLE STRANDED DNA; SMN1 PROTEIN, HUMAN; SMN2 PROTEIN, HUMAN; SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN 2;

EID: 80855144747     PISSN: 09628819     EISSN: None     Source Type: Journal    
DOI: 10.1007/s11248-011-9496-8     Document Type: Article
Times cited : (42)

References (51)
  • 3
    • 33745144314 scopus 로고    scopus 로고
    • Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector
    • Baughan T, Shababi M, Coady TH, Dickson AM, Tullis GE, Lorson CL (2006) Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. Mol Ther 14: 54-62.
    • (2006) Mol Ther , vol.14 , pp. 54-62
    • Baughan, T.1    Shababi, M.2    Coady, T.H.3    Dickson, A.M.4    Tullis, G.E.5    Lorson, C.L.6
  • 4
    • 64549161948 scopus 로고    scopus 로고
    • Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy
    • Baughan TD, Dickson A, Osman EY, Lorson CL (2009) Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy. Hum Mol Genet 18: 1600-1611.
    • (2009) Hum Mol Genet , vol.18 , pp. 1600-1611
    • Baughan, T.D.1    Dickson, A.2    Osman, E.Y.3    Lorson, C.L.4
  • 7
    • 0036544654 scopus 로고    scopus 로고
    • Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1
    • Cartegni L, Krainer AR (2002) Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1. Nat Genet 30: 377-384.
    • (2002) Nat Genet , vol.30 , pp. 377-384
    • Cartegni, L.1    Krainer, A.R.2
  • 9
    • 74849129866 scopus 로고    scopus 로고
    • Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy
    • Coady TH, Lorson CL (2010) Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy. J Neurosci 30: 126-130.
    • (2010) J Neurosci , vol.30 , pp. 126-130
    • Coady, T.H.1    Lorson, C.L.2
  • 10
    • 55649102490 scopus 로고    scopus 로고
    • Development of a single vector system that enhances trans-splicing of SMN2 transcripts
    • Coady TH, Baughan TD, Shababi M, Passini MA, Lorson CL (2008) Development of a single vector system that enhances trans-splicing of SMN2 transcripts. PLoS One 3: e3468.
    • (2008) PLoS One , vol.3
    • Coady, T.H.1    Baughan, T.D.2    Shababi, M.3    Passini, M.A.4    Lorson, C.L.5
  • 15
    • 77949519556 scopus 로고    scopus 로고
    • Restoration of full-length SMN promoted by adenoviral vectors expressing RNA antisense oligonucleotides embedded in U7 snRNAs
    • Geib T, Hertel KJ (2009) Restoration of full-length SMN promoted by adenoviral vectors expressing RNA antisense oligonucleotides embedded in U7 snRNAs. PLoS One 4: e8204.
    • (2009) PLoS One , vol.4
    • Geib, T.1    Hertel, K.J.2
  • 18
    • 41549168514 scopus 로고    scopus 로고
    • Antisense masking of an hnRNP A1/A2 intronic splice silencer corrects SMN2 splicing in transgenic mice
    • Hua Y, Vickers T, Okunola H, Bennett C, Krainer, A (2008) Antisense masking of an hnRNP A1/A2 intronic splice silencer corrects SMN2 splicing in transgenic mice. Am J Hum Genet 82: 834-848.
    • (2008) Am J Hum Genet , vol.82 , pp. 834-848
    • Hua, Y.1    Vickers, T.2    Okunola, H.3    Bennett, C.4    Krainer, A.5
  • 19
    • 77955894067 scopus 로고    scopus 로고
    • Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
    • Hua Y, Sahaski K, Hung G, Rigo F, Passini M, Bennett C, Krainer A (2010) Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev 24: 1634-1644.
    • (2010) Genes Dev , vol.24 , pp. 1634-1644
    • Hua, Y.1    Sahaski, K.2    Hung, G.3    Rigo, F.4    Passini, M.5    Bennett, C.6    Krainer, A.7
  • 20
    • 4344627003 scopus 로고    scopus 로고
    • Creating genetically modified pigs by using nuclear transfer
    • Lai L, Prather RS (2003) Creating genetically modified pigs by using nuclear transfer. Reprod Biol Endocrinol 1: 82.
    • (2003) Reprod Biol Endocrinol , vol.1 , pp. 82
    • Lai, L.1    Prather, R.S.2
  • 22
    • 20144385587 scopus 로고    scopus 로고
    • SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN
    • Le TT, Pham LT, Butchbach ME, Zhang HL, Monani UR, Coovert DD, Gavrilina TO, Xing L, Bassell GJ, Burghes AH (2005) SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN. Hum Mol Genet 14: 845-857.
    • (2005) Hum Mol Genet , vol.14 , pp. 845-857
    • Le, T.T.1    Pham, L.T.2    Butchbach, M.E.3    Zhang, H.L.4    Monani, U.R.5    Coovert, D.D.6    Gavrilina, T.O.7    Xing, L.8    Bassell, G.J.9    Burghes, A.H.10
  • 23
    • 0035976992 scopus 로고    scopus 로고
    • Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3′ splice site pairing
    • Lim SR, Hertel KJ (2001) Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3′ splice site pairing. J Biol Chem 276: 45476-45483.
    • (2001) J Biol Chem , vol.276 , pp. 45476-45483
    • Lim, S.R.1    Hertel, K.J.2
  • 24
    • 0033033434 scopus 로고    scopus 로고
    • A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy
    • Lorson CL, Hahnen E, Androphy EJ, Wirth B (1999) A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Proc Natl Acad Sci USA 96: 6307-6311.
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 6307-6311
    • Lorson, C.L.1    Hahnen, E.2    Androphy, E.J.3    Wirth, B.4
  • 25
    • 49449101788 scopus 로고    scopus 로고
    • Identification and characterization of the porcine (Sus scrofa) survival motor neuron (SMN1) gene: an animal model for therapeutic studies
    • Lorson MA, Spate LD, Prather RS, Lorson CL (2008) Identification and characterization of the porcine (Sus scrofa) survival motor neuron (SMN1) gene: an animal model for therapeutic studies. Dev Dyn 237: 2268-2278.
    • (2008) Dev Dyn , vol.237 , pp. 2268-2278
    • Lorson, M.A.1    Spate, L.D.2    Prather, R.S.3    Lorson, C.L.4
  • 26
    • 77953893282 scopus 로고    scopus 로고
    • Spinal muscular atrophy: mechanisms and therapeutic strategies
    • Lorson CL, Rindt H, Shababi M (2010) Spinal muscular atrophy: mechanisms and therapeutic strategies. Hum Mol Genet 19: 111-118.
    • (2010) Hum Mol Genet , vol.19 , pp. 111-118
    • Lorson, C.L.1    Rindt, H.2    Shababi, M.3
  • 27
    • 0030862402 scopus 로고    scopus 로고
    • Complete activation of porcine oocytes induced by the sulfhydryl reagent, thimerosal
    • Machaty Z, Wang WH, Day BN, Prather RS (1997) Complete activation of porcine oocytes induced by the sulfhydryl reagent, thimerosal. Biol Reprod 57: 1123-1127.
    • (1997) Biol Reprod , vol.57 , pp. 1123-1127
    • Machaty, Z.1    Wang, W.H.2    Day, B.N.3    Prather, R.S.4
  • 28
    • 77749298729 scopus 로고    scopus 로고
    • Genetic therapy for spinal muscular atrophy
    • MacKenzie A (2010) Genetic therapy for spinal muscular atrophy. Nat Biotechnol 28: 235-237.
    • (2010) Nat Biotechnol , vol.28 , pp. 235-237
    • Mackenzie, A.1
  • 29
    • 28444490312 scopus 로고    scopus 로고
    • Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs
    • Madocsai C, Lim SR, Geib T, Lam BJ, Hertel KJ (2005) Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs. Mol Ther 12: 1013-1022.
    • (2005) Mol Ther , vol.12 , pp. 1013-1022
    • Madocsai, C.1    Lim, S.R.2    Geib, T.3    Lam, B.J.4    Hertel, K.J.5
  • 30
    • 33750570039 scopus 로고    scopus 로고
    • Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts
    • Mattis VB, Rai R, Wang J, Chang CW, Coady T, Lorson CL (2006) Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts. Hum Genet 120: 589-601.
    • (2006) Hum Genet , vol.120 , pp. 589-601
    • Mattis, V.B.1    Rai, R.2    Wang, J.3    Chang, C.W.4    Coady, T.5    Lorson, C.L.6
  • 31
    • 70349575755 scopus 로고    scopus 로고
    • Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model
    • Mattis VB, Ebert AD, Fosso MY, Chang CW, Lorson CL (2009a) Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model. Hum Mol Genet 18: 3906-3913.
    • (2009) Hum Mol Genet , vol.18 , pp. 3906-3913
    • Mattis, V.B.1    Ebert, A.D.2    Fosso, M.Y.3    Chang, C.W.4    Lorson, C.L.5
  • 32
    • 71949095416 scopus 로고    scopus 로고
    • Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA
    • Mattis VB, Fosso MY, Chang CW, Lorson CL (2009b) Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA. BMC Neurosci 10: 142.
    • (2009) BMC Neurosci , vol.10 , pp. 142
    • Mattis, V.B.1    Fosso, M.Y.2    Chang, C.W.3    Lorson, C.L.4
  • 38
    • 78650115906 scopus 로고    scopus 로고
    • Perspectives and diagnostic considerations in spinal muscular atrophy
    • Prior TW (2010) Perspectives and diagnostic considerations in spinal muscular atrophy. Genet Med 12: 145-152.
    • (2010) Genet Med , vol.12 , pp. 145-152
    • Prior, T.W.1
  • 41
    • 77954542506 scopus 로고    scopus 로고
    • Optimization of square-wave electroporation for transfection of porcine fetal fibroblasts
    • Ross JW, Whyte JJ, Zhao J, Samuel M, Wells KD, Prather RS (2010) Optimization of square-wave electroporation for transfection of porcine fetal fibroblasts. Transgenic Res 19: 611-620.
    • (2010) Transgenic Res , vol.19 , pp. 611-620
    • Ross, J.W.1    Whyte, J.J.2    Zhao, J.3    Samuel, M.4    Wells, K.D.5    Prather, R.S.6
  • 42
    • 0030931720 scopus 로고    scopus 로고
    • Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos
    • Schrank B, Gotz R, Gunnersen JM, Ure JM, Toyka KV, Smith AG, Sendtner M (1997) Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos. Proc Natl Acad Sci USA 94: 9920-9925.
    • (1997) Proc Natl Acad Sci USA , vol.94 , pp. 9920-9925
    • Schrank, B.1    Gotz, R.2    Gunnersen, J.M.3    Ure, J.M.4    Toyka, K.V.5    Smith, A.G.6    Sendtner, M.7
  • 43
    • 67650480122 scopus 로고    scopus 로고
    • A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy
    • Singh NN, Shishimorova M, Cao LC, Gangwani L, Singh RN (2009) A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy. RNA Biol 6: 341-350.
    • (2009) RNA Biol , vol.6 , pp. 341-350
    • Singh, N.N.1    Shishimorova, M.2    Cao, L.C.3    Gangwani, L.4    Singh, R.N.5
  • 44
    • 77950462461 scopus 로고    scopus 로고
    • Spinal muscular atrophy disease: a literature review for therapeutic strategies
    • Stavarachi M, Apostol P, Toma M, Cimponeriu D, Gavrila L (2010) Spinal muscular atrophy disease: a literature review for therapeutic strategies. J Med Life 3: 3-9.
    • (2010) J Med Life , vol.3 , pp. 3-9
    • Stavarachi, M.1    Apostol, P.2    Toma, M.3    Cimponeriu, D.4    Gavrila, L.5
  • 46
    • 39149084412 scopus 로고    scopus 로고
    • Synthesis and biological evaluation of novel 2, 4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy
    • Thurmond J, Butchbach ME, Palomo M, Pease B, Rao M, Bedell L, Keyvan M, Pai G, Mishra R, Haraldsson M et al (2008) Synthesis and biological evaluation of novel 2, 4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy. J Med Chem 51: 449-469.
    • (2008) J Med Chem , vol.51 , pp. 449-469
    • Thurmond, J.1    Butchbach, M.E.2    Palomo, M.3    Pease, B.4    Rao, M.5    Bedell, L.6    Keyvan, M.7    Pai, G.8    Mishra, R.9    Haraldsson, M.10
  • 48
    • 0034007548 scopus 로고    scopus 로고
    • An update of the mutation spectrum of the survival motor neuron gene (SMN1) in autosomal recessive spinal muscular atrophy (SMA)
    • Wirth B (2000) An update of the mutation spectrum of the survival motor neuron gene (SMN1) in autosomal recessive spinal muscular atrophy (SMA). Hum Mutat 15: 228-237.
    • (2000) Hum Mutat , vol.15 , pp. 228-237
    • Wirth, B.1
  • 49
    • 18144431041 scopus 로고    scopus 로고
    • A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels
    • Wolstencroft EC, Mattis V, Bajer AA, Young PJ, Lorson CL (2005) A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels. Hum Mol Genet 14: 1199-1210.
    • (2005) Hum Mol Genet , vol.14 , pp. 1199-1210
    • Wolstencroft, E.C.1    Mattis, V.2    Bajer, A.A.3    Young, P.J.4    Lorson, C.L.5
  • 50
    • 0035158371 scopus 로고    scopus 로고
    • An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA
    • Zhang ML, Lorson CL, Androphy EJ, Zhou J (2001) An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA. Gene Ther 8: 1532-1538.
    • (2001) Gene Ther , vol.8 , pp. 1532-1538
    • Zhang, M.L.1    Lorson, C.L.2    Androphy, E.J.3    Zhou, J.4
  • 51
    • 69849091474 scopus 로고    scopus 로고
    • Significant improvement in cloning efficiency of an inbred miniature pig by histone deacetylase inhibitor treatment after somatic cell nuclear transfer
    • Zhao J, Ross JW, Hao Y, Spate LD, Walters EM, Samuel MS, Rieke A, Murphy CN, Prather RS (2009) Significant improvement in cloning efficiency of an inbred miniature pig by histone deacetylase inhibitor treatment after somatic cell nuclear transfer. Biol Reprod 81: 525-530.
    • (2009) Biol Reprod , vol.81 , pp. 525-530
    • Zhao, J.1    Ross, J.W.2    Hao, Y.3    Spate, L.D.4    Walters, E.M.5    Samuel, M.S.6    Rieke, A.7    Murphy, C.N.8    Prather, R.S.9


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.