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Volumn 37, Issue 2, 2015, Pages 209-215

Correction of a cystic fibrosis splicing mutation by antisense oligonucleotides

Author keywords

Antisense oligonucleotide; CFTR; Cystic fibrosis; Exon skipping; Splicing mutation

Indexed keywords

ERRATUM; CYSTIC FIBROSIS; DNA SEQUENCE; EXON; GENE THERAPY; GENETICS; HEK293 CELL LINE; HIGH THROUGHPUT SEQUENCING; HUMAN; INTRON; METABOLISM; MOLECULAR GENETICS; MUTATION; NUCLEOTIDE SEQUENCE; PATHOLOGY; RNA SPLICING;

EID: 85015660898     PISSN: 10597794     EISSN: 10981004     Source Type: Journal    
DOI: 10.1002/humu.22931     Document Type: Erratum
Times cited : (59)

References (31)
  • 1
    • 84921487109 scopus 로고    scopus 로고
    • Novel personalized therapies for cystic fibrosis: Treating the basic defect in all patients
    • Amaral MD. 2015. Novel personalized therapies for cystic fibrosis: treating the basic defect in all patients. J Intern Med 277:155–166.
    • (2015) J Intern Med , vol.277 , pp. 155-166
    • Amaral, M.D.1
  • 2
    • 0141681935 scopus 로고    scopus 로고
    • EGCG corrects aberrant splicing of IKAP mRNA in cells frompatients with familial dysautonomia
    • Anderson SL, Qiu J, Rubin BY. 2003. EGCG corrects aberrant splicing of IKAP mRNA in cells frompatients with familial dysautonomia. Biochem Biophys Res Commun 310:627–633.
    • (2003) Biochem Biophys Res Commun , vol.310 , pp. 627-633
    • Anderson, S.L.1    Qiu, J.2    Rubin, B.Y.3
  • 3
    • 84918819788 scopus 로고    scopus 로고
    • New pharmacological approaches for cystic fibrosis: Promises, progress, pitfalls
    • Bell SC, De Boeck K, Amaral MD. 2015. New pharmacological approaches for cystic fibrosis: promises, progress, pitfalls. Pharmacol Ther 145:19–34.
    • (2015) Pharmacol Ther , vol.145 , pp. 19-34
    • Bell, S.C.1    De Boeck, K.2    Amaral, M.D.3
  • 5
    • 0035282581 scopus 로고    scopus 로고
    • Aberrant CFTR-dependent HCO3-transport in mutations associated with cystic fibrosis
    • Choi JY, Muallem D, Kiselyov K, Lee MG, Thomas PJ, Muallem S. 2001. Aberrant CFTR-dependent HCO3-transport in mutations associated with cystic fibrosis. Nature 410(6824):94–97.
    • (2001) Nature , vol.410 , Issue.6824 , pp. 94-97
    • Choi, J.Y.1    Muallem, D.2    Kiselyov, K.3    Lee, M.G.4    Thomas, P.J.5    Muallem, S.6
  • 6
    • 0026656343 scopus 로고
    • Extensive posttranscriptional deletion of the coding sequences for part of nucleotide-binding fold 1 in respiratory epithelial mRNA transcripts of the cystic fibrosis transmembrane conductance regulator gene is not associated with the clinical manifestations of cystic fibrosis
    • Chu CS, Trapnell BC, Curristin SM, Cutting GR, Crystal RG. 1992. Extensive posttranscriptional deletion of the coding sequences for part of nucleotide-binding fold 1 in respiratory epithelial mRNA transcripts of the cystic fibrosis transmembrane conductance regulator gene is not associated with the clinical manifestations of cystic fibrosis. J Clin Invest 90:785–790.
    • (1992) J Clin Invest , vol.90 , pp. 785-790
    • Chu, C.S.1    Trapnell, B.C.2    Curristin, S.M.3    Cutting, G.R.4    Crystal, R.G.5
  • 8
    • 84920733762 scopus 로고    scopus 로고
    • Improvement of SMN2pre-mRNA processing mediated by exon-specific U1 small nuclear RNA
    • DalMas A, Rogalska ME, Bussani E, Pagani F. 2015. Improvement of SMN2pre-mRNA processing mediated by exon-specific U1 small nuclear RNA. Am J Hum Genet 96:93–103.
    • (2015) Am J Hum Genet , vol.96 , pp. 93-103
    • Dalmas, A.1    Rogalska, M.E.2    Bussani, E.3    Pagani, F.4
  • 15
    • 84856431819 scopus 로고    scopus 로고
    • RNA therapeutics: Beyond RNA interference and antisense oligonucleotides
    • Kole R, Krainer AR, Altman S. 2012. RNA therapeutics: beyond RNA interference and antisense oligonucleotides. Nat Rev Drug Discov 11:125–140.
    • (2012) Nat Rev Drug Discov , vol.11 , pp. 125-140
    • Kole, R.1    Krainer, A.R.2    Altman, S.3
  • 19
    • 84880030599 scopus 로고    scopus 로고
    • Management of comorbidities in older patients with cystic fibrosis
    • Plant BJ, Goss CH, Plant WD, Bell SC. 2013. Management of comorbidities in older patients with cystic fibrosis. Lancet Respir Med 1:164–174.
    • (2013) Lancet Respir Med , vol.1 , pp. 164-174
    • Plant, B.J.1    Goss, C.H.2    Plant, W.D.3    Bell, S.C.4
  • 20
    • 0036846329 scopus 로고    scopus 로고
    • Five percent of normal cystic fibrosis transmembrane conductance regulator mRNA ameliorates the severity of pulmonary disease in cystic fibrosis
    • Ramalho A.S, Beck S, Meyer M, Penque D, Cutting GR, Amaral MD.2002. Five percent of normal cystic fibrosis transmembrane conductance regulator mRNA ameliorates the severity of pulmonary disease in cystic fibrosis. Am J Respir Cell Mol Biol 27:619–627.
    • (2002) Am J Respir Cell Mol Biol , vol.27 , pp. 619-627
    • Ramalho, A.S.1    Beck, S.2    Meyer, M.3    Penque, D.4    Cutting, G.R.5    Amaral, M.D.6
  • 21
    • 85051362213 scopus 로고    scopus 로고
    • Comparative ex vivo, in vitro and in silico analyses of a CFTR splicing mutation: Importance of functional studies to establish disease liability of mutations
    • Ramalho AS, Clarke LA, Sousa M, Felicio V, Barreto C, Lopes C, Amaral MD. 2015. Comparative ex vivo, in vitro and in silico analyses of a CFTR splicing mutation: Importance of functional studies to establish disease liability of mutations. J Cyst Fibros.[Epub ahead of print].
    • (2015) J Cyst Fibros
    • Ramalho, A.S.1    Clarke, L.A.2    Sousa, M.3    Felicio, V.4    Barreto, C.5    Lopes, C.6    Amaral, M.D.7
  • 22
    • 84873022115 scopus 로고    scopus 로고
    • Pick one, but be quick: 5’ splice sites and the problems of too many choices
    • Roca X, Krainer AR, Eperon IC. 2013. Pick one, but be quick: 5’ splice sites and the problems of too many choices. Genes Dev 27:129–144.
    • (2013) Genes Dev , vol.27 , pp. 129-144
    • Roca, X.1    Krainer, A.R.2    Eperon, I.C.3
  • 24
    • 32044445564 scopus 로고    scopus 로고
    • Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron
    • Singh NK, Singh NN, Androphy EJ, Singh RN. 2006. Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron. Mol Cell Biol 26:1333–1346.
    • (2006) Mol Cell Biol , vol.26 , pp. 1333-1346
    • Singh, N.K.1    Singh, N.N.2    Rophy, E.J.3    Singh, R.N.4
  • 26
    • 84858405200 scopus 로고    scopus 로고
    • Splice modulating therapies for human disease
    • Spitali P, Aartsma-Rus A. 2012. Splice modulating therapies for human disease. Cell 148:1085–1088.
    • (2012) Cell , vol.148 , pp. 1085-1088
    • Spitali, P.1    Aartsma-Rus, A.2
  • 27
    • 49649108878 scopus 로고    scopus 로고
    • A high-throughput screening strategy identifies cardiotonic steroids as alternative splicing modulators
    • Stoilov P, Lin CH, Damoiseaux R, Nikolic J, Black DL. 2008. A high-throughput screening strategy identifies cardiotonic steroids as alternative splicing modulators. Proc Natl Acad Sci USA 105:11218–11223.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 11218-11223
    • Stoilov, P.1    Lin, C.H.2    Damoiseaux, R.3    Nikolic, J.4    Black, D.L.5
  • 28
    • 45249091505 scopus 로고    scopus 로고
    • Substances that can change alternative splice-site selection
    • Sumanasekera C, Watt DS, Stamm S. 2008. Substances that can change alternative splice-site selection. Biochem Soc Trans 36(Pt 3):483–490.
    • (2008) Biochem Soc Trans , vol.36 , Issue.3 , pp. 483-490
    • Sumanasekera, C.1    Watt, D.S.2    Stamm, S.3
  • 29
    • 84900835509 scopus 로고    scopus 로고
    • New developments in exon skipping and splice modulation therapies for neuromuscular diseases
    • Touznik A, Lee JJ, Yokota T. 2014. New developments in exon skipping and splice modulation therapies for neuromuscular diseases. Expert Opin Biol Ther 14:809–819.
    • (2014) Expert Opin Biol Ther , vol.14 , pp. 809-819
    • Touznik, A.1    Lee, J.J.2    Yokota, T.3
  • 30
    • 60349104299 scopus 로고    scopus 로고
    • The spliceosome: Design principles of a dynamic RNP machine
    • Wahl MC, Will C.L, Luhrmann R. 2009. The spliceosome: design principles of a dynamic RNP machine. Cell 136:701–718.
    • (2009) Cell , vol.136 , pp. 701-718
    • Wahl, M.C.1    Will, C.L.2    Luhrmann, R.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.