Abstract
Spliceosome-mediated RNA trans-splicing (SMaRT) is a tool that facilitates the recombination of two distinct pre-mRNA molecules. Its application for gene therapeutic purposes has been hindered by laborious procedures to identify gene-specific molecules. We have established a screening method for the identification of highly functional RNA trans-splicing molecules based on fluorescence reporters, facilitating the generation of most potent therapeutic molecules for the correction of any gene of interest.
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References
Wally V, Brunner M, Lettner T, Wagner M, Koller U, Trost A, Murauer EM, Hainzl S, Hintner H, Bauer JW (2010) K14 mRNA reprogramming for dominant epidermolysis bullosa simplex. Hum Mol Genet 19:4715–4725
Gruber C, Gratz IK, Murauer EM, Mayr E, Koller U, Bruckner-Tuderman L, Meneguzzi G, Hintner H, Bauer JW (2011) Spliceosome-mediated RNA trans-splicing facilitates targeted delivery of suicide genes to cancer cells. Mol Cancer Ther 10:233–241
Murauer EM, Gache Y, Gratz IK, Klausegger A, Muss W, Gruber C, Meneguzzi G, Hintner H, Bauer JW (2011) Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa. J Invest Dermatol 131:74–83
Yang YP, Walsh CE (2005) Spliceosome-mediated RNA trans-splicing. Mol Ther 12:1006–1012
Koller U, Wally V, Mitchell LG, Klausegger A, Murauer EM, Mayr E, Gruber C, Hainzl S, Hintner H, Bauer JW (2011) A novel screening system improves genetic correction by internal exon replacement. Nucleic Acids Res 39:e108
Coady TH, Lorson CL (2010) Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy. J Neurosci 30:126–130
Shababi M, Glascock J, Lorson CL (2010) Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy. Hum Gene Ther 22:135–144
Rodriguez-Martin T, Anthony K, Garcia-Blanco MA, Mansfield SG, Anderton BH, Gallo JM (2009) Correction of tau mis-splicing caused by FTDP-17 MAPT mutations by spliceosome-mediated RNA trans-splicing. Hum Mol Genet 18:3266–3273
Song Y, Lou HH, Boyer JL, Limberis MP, Vandenberghe LH, Hackett NR, Leopold PL, Wilson JM, Crystal RG (2009) Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing. Hum Gene Ther 20:267–281
Wally V, Klausegger A, Koller U, Lochmuller H, Krause S, Wiche G, Mitchell LG, Hintner H, Bauer JW (2008) 5′ trans-splicing repair of the PLEC1 gene. J Invest Dermatol 128:568–574
Sambrook J, Russel DW (2001) Molecular cloning: a laboratory manual. Cold Spring Harbor Laboratory Press, New York
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Bauer, J.W., Murauer, E.M., Wally, V., Koller, U. (2013). RNA Trans-Splicing for Genodermatoses. In: Has, C., Sitaru, C. (eds) Molecular Dermatology. Methods in Molecular Biology, vol 961. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-62703-227-8_30
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DOI: https://doi.org/10.1007/978-1-62703-227-8_30
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Publisher Name: Humana Press, Totowa, NJ
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