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Volumn 16, Issue 1, 2015, Pages 1179-1191

Trans-Splicing improvement by the combined application of Antisense strategies

Author keywords

Antisense oligonucleotides; Genetic diseases; RNA repair; RNA trans splicing

Indexed keywords

COMPLEMENTARY RNA; GREEN FLUORESCENT PROTEIN; ANTISENSE OLIGONUCLEOTIDE; COLLAGEN TYPE 7;

EID: 84920546316     PISSN: 16616596     EISSN: 14220067     Source Type: Journal    
DOI: 10.3390/ijms16011179     Document Type: Article
Times cited : (16)

References (25)
  • 6
    • 84863983970 scopus 로고    scopus 로고
    • Spliceosome-mediated trans-splicing: The therapeutic cut and paste
    • Wally, V.; Murauer, E.M.; Bauer, J.W. Spliceosome-mediated trans-splicing: The therapeutic cut and paste. J. Investig. Dermatol. 2012, 132, 1959-1966.
    • (2012) J. Investig. Dermatol , vol.132 , pp. 1959-1966
    • Wally, V.1    Murauer, E.M.2    Bauer, J.W.3
  • 7
    • 77952527820 scopus 로고    scopus 로고
    • Gene therapy for recessive dystrophic epidermolysis bullosa
    • Titeux, M.; Pendaries, V.; Hovnanian, A. Gene therapy for recessive dystrophic epidermolysis bullosa. Dermatol. Clin. 2010, 28, 361-366.
    • (2010) Dermatol. Clin , vol.28 , pp. 361-366
    • Titeux, M.1    Pendaries, V.2    Hovnanian, A.3
  • 9
    • 84878299265 scopus 로고    scopus 로고
    • Dijana Plaseska-Karanfilska, Ed.; InTech: Shanghai, China
    • Wally, V.; Koller, U.; Bauer, J.W. Human Genetic Diseases; Dijana Plaseska-Karanfilska, Ed.; InTech: Shanghai, China, 2011; pp. 223-240.
    • (2011) Human Genetic Diseases , pp. 223-240
    • Wally, V.1    Koller, U.2    Bauer, J.W.3
  • 10
    • 84878329835 scopus 로고    scopus 로고
    • A reporter-based screen to identify potent 3' trans-splicing molecules for endogenous RNA repair
    • Murauer, E.M.; Koller, U.; Hainzl, S.; Wally, V.; Bauer, J.W. A reporter-based screen to identify potent 3' trans-splicing molecules for endogenous RNA repair. Hum. Gene Ther. Methods 2013, 24, 19-27.
    • (2013) Hum. Gene Ther. Methods , vol.24 , pp. 19-27
    • Murauer, E.M.1    Koller, U.2    Hainzl, S.3    Wally, V.4    Bauer, J.W.5
  • 11
    • 84920517499 scopus 로고    scopus 로고
    • Considerations for a successful RNA trans-splicing repair of genetic disorders
    • Koller, U.; Wally, V.; Bauer, J.W.; Murauer, E.M. Considerations for a successful RNA trans-splicing repair of genetic disorders. Mol. Ther. Nucleic Acids 2014, 3, e157.
    • (2014) Mol. Ther. Nucleic Acids , vol.3
    • Koller, U.1    Wally, V.2    Bauer, J.W.3    Murauer, E.M.4
  • 13
    • 55649102490 scopus 로고    scopus 로고
    • Development of a single vector system that enhances trans-splicing of SMN2 transcripts
    • Coady, T.H.; Baughan, T.D.; Shababi, M.; Passini, M.A.; Lorson, C.L. Development of a single vector system that enhances trans-splicing of SMN2 transcripts. PLoS One 2008, 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
  • 14
    • 74849129866 scopus 로고    scopus 로고
    • Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy
    • Coady, T.H.; Lorson, C.L. Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy. J. Neurosci. 2010, 30, 126-130.
    • (2010) J. Neurosci , vol.30 , pp. 126-130
    • Coady, T.H.1    Lorson, C.L.2
  • 15
    • 41549168514 scopus 로고    scopus 로고
    • Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice
    • Hua, Y.; Vickers, T.A.; Okunola, H.L.; Bennett, C.F.; Krainer, A.R. Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice. Am. J. Hum. Genet. 2008, 82, 834-848.
    • (2008) Am. J. Hum. Genet , vol.82 , pp. 834-848
    • Hua, Y.1    Vickers, T.A.2    Okunola, H.L.3    Bennett, C.F.4    Krainer, A.R.5
  • 21
    • 27144477400 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: Reprograming gene expression by trans-splicing
    • Mitchell, L.G.; McGarrity, G.J. Gene therapy progress and prospects: Reprograming gene expression by trans-splicing. Gene Ther. 2005, 12, 1477-1485.
    • (2005) Gene Ther , vol.12 , pp. 1477-1485
    • Mitchell, L.G.1    McGarrity, G.J.2
  • 22
    • 0021802802 scopus 로고
    • Evaluation of anchoring fibrils and other components of the dermal-epidermal junction in dystrophic epidermolysis bullosa by a quantitative ultrastructural technique
    • Tidman, M.J.; Eady, R.A. Evaluation of anchoring fibrils and other components of the dermal-epidermal junction in dystrophic epidermolysis bullosa by a quantitative ultrastructural technique. J. Investig. Dermatol. 1985, 84, 374-377.
    • (1985) J. Investig. Dermatol. , vol.84 , pp. 374-377
    • Tidman, M.J.1    Eady, R.A.2
  • 24
    • 71049190003 scopus 로고    scopus 로고
    • Dominant negative effects of COL7A1 mutations can be rescued by controlled over-expression of normal collagen VII
    • Fritsch, A.; Spassov, S.; Elfert, S.; Schlosser, A.; Gache, Y.; Meneguzzi, G.; Bruckner-Tuderman, L. Dominant negative effects of COL7A1 mutations can be rescued by controlled over-expression of normal collagen VII. J. Biol. Chem. 2009, 284, 30248-30256.
    • (2009) J. Biol. Chem , vol.284 , pp. 30248-30256
    • Fritsch, A.1    Spassov, S.2    Elfert, S.3    Schlosser, A.4    Gache, Y.5    Meneguzzi, G.6    Bruckner-Tuderman, L.7
  • 25
    • 0035809198 scopus 로고    scopus 로고
    • An inducible mouse model for epidermolysis bullosa simplex: Implications for gene therapy
    • Cao, T.Y.; Longley, M.A.; Wang, X.J.; Roop, D.R. An inducible mouse model for epidermolysis bullosa simplex: Implications for gene therapy. J. Cell Biol. 2001, 152, 651-656.
    • (2001) J. Cell Biol , vol.152 , pp. 651-656
    • Cao, T.Y.1    Longley, M.A.2    Wang, X.J.3    Roop, D.R.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.