-
2
-
-
2942525218
-
Molecular imaging of gene expression in living subjects by spliceosome-mediated RNA trans-splicing
-
Bhaumik S, Walls Z, Puttaraju M et al. (2004) Molecular imaging of gene expression in living subjects by spliceosome-mediated RNA trans-splicing. Proc Natl Acad Sci USA 101:8693-8
-
(2004)
Proc Natl Acad Sci USA
, vol.101
, pp. 8693-8698
-
-
Bhaumik, S.1
Walls, Z.2
Puttaraju, M.3
-
3
-
-
0031787859
-
Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice
-
Brown MP, Topham DJ, Sangster MY et al. (1998) Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice. Nat Med 4:1253-60
-
(1998)
Nat Med
, vol.4
, pp. 1253-1260
-
-
Brown, M.P.1
Topham, D.J.2
Sangster, M.Y.3
-
4
-
-
0141743751
-
Efficient and specific repair of sickle beta-globin RNA by trans-splicing ribozymes
-
Byun J, Lan N, Long M et al. (2003) Efficient and specific repair of sickle beta-globin RNA by trans-splicing ribozymes. RNA 9:1254-63
-
(2003)
RNA
, vol.9
, pp. 1254-1263
-
-
Byun, J.1
Lan, N.2
Long, M.3
-
5
-
-
0035809198
-
An inducible mouse model for epidermolysis bullosa simplex: Implications for gene therapy
-
Cao TY, Longley MA, Wang XJ et al. (2001) An inducible mouse model for epidermolysis bullosa simplex: implications for gene therapy. J Cell Biol 152:651-6
-
(2001)
J Cell Biol
, vol.152
, pp. 651-656
-
-
Cao, T.Y.1
Longley, M.A.2
Wang, X.J.3
-
6
-
-
0042388694
-
Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing
-
Chao HJ, Mansfield SG, Bartel RC et al. (2003) Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing. Nat Med 9:1015-9
-
(2003)
Nat Med
, vol.9
, pp. 1015-1019
-
-
Chao, H.J.1
Mansfield, S.G.2
Bartel, R.C.3
-
7
-
-
55649102490
-
Development of a single vector system that enhances trans-splicing of SMN2 transcripts
-
Coady TH, Baughan TD, Shababi M et al. (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
-
8
-
-
74849129866
-
Trans-splicingmediated improvement in a severe mouse model of spinal muscular atrophy
-
Coady TH, Lorson CL (2010) Trans-splicingmediated improvement in a severe mouse model of spinal muscular atrophy. J Neurosci 30:126-30
-
(2010)
J Neurosci
, vol.30
, pp. 126-130
-
-
Coady, T.H.1
Lorson, C.L.2
-
9
-
-
34547117851
-
Restoration of SMN function: Delivery of a trans-splicing RNA re-directs SMN2 premRNA splicing
-
Coady TH, Shababi M, Tullis GE et al. (2007) Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 premRNA splicing. Mol Ther 15:1471-8
-
(2007)
Mol Ther
, vol.15
, pp. 1471-1478
-
-
Coady, T.H.1
Shababi, M.2
Tullis, G.E.3
-
10
-
-
0043175257
-
Development of spliceosomemediated RNA trans-splicing (SMaRT) for the correction of inherited skin diseases
-
Dallinger G, Puttaraju M, Mitchell LG et al. (2003) Development of spliceosomemediated RNA trans-splicing (SMaRT) for the correction of inherited skin diseases. Exp Dermatol 12:37-46
-
(2003)
Exp Dermatol
, vol.12
, pp. 37-46
-
-
Dallinger, G.1
Puttaraju, M.2
Mitchell, L.G.3
-
11
-
-
0029143929
-
RNA trans-splicing in flatworms. Analysis of trans-spliced mRNAs and genes in the human parasite, Schistosoma mansoni
-
Davis RE, Hardwick C, Tavernier P et al. (1995) RNA trans-splicing in flatworms. Analysis of trans-spliced mRNAs and genes in the human parasite, Schistosoma mansoni. J Biol Chem 270:21813-9
-
(1995)
J Biol Chem
, vol.270
, pp. 21813-21819
-
-
Davis, R.E.1
Hardwick, C.2
Tavernier, P.3
-
12
-
-
77952880881
-
RNA reprogramming and repair based on trans-splicing group i ribozymes
-
Fiskaa T, Birgisdottir AB (2010) RNA reprogramming and repair based on trans-splicing group I ribozymes. New Biotechnol 27: 194-203
-
(2010)
New Biotechnol
, vol.27
, pp. 194-203
-
-
Fiskaa, T.1
Birgisdottir, A.B.2
-
13
-
-
0037135521
-
Natural trans-spliced mRNAs are generated from the human estrogen receptor-alpha (hER alpha) gene
-
Flouriot G, Brand H, Seraphin B et al. (2002) Natural trans-spliced mRNAs are generated from the human estrogen receptor-alpha (hER alpha) gene. J Biol Chem 277:26244-51
-
(2002)
J Biol Chem
, vol.277
, pp. 26244-26251
-
-
Flouriot, G.1
Brand, H.2
Seraphin, B.3
-
14
-
-
0015492624
-
Gene therapy for human genetic disease?
-
Friedmann T, Roblin R (1972) Gene therapy for human genetic disease? Science 175:949-55
-
(1972)
Science
, vol.175
, pp. 949-955
-
-
Friedmann, T.1
Roblin, R.2
-
15
-
-
71049190003
-
Dominant negative effects of COL7A1 mutations can be rescued by controlled overexpression of normal collagen VII
-
Fritsch A, Spassov S, Elfert S et al. (2009) Dominant negative effects of COL7A1 mutations can be rescued by controlled overexpression of normal collagen VII. J Biol Chem 284:30248-56
-
(2009)
J Biol Chem
, vol.284
, pp. 30248-30256
-
-
Fritsch, A.1
Spassov, S.2
Elfert, S.3
-
16
-
-
79951834142
-
Spliceosome-mediated RNA trans-splicing facilitates targeted delivery of suicide genes to cancer cells
-
Gruber C, Gratz IK, Murauer EM et al. (2011) Spliceosome-mediated RNA trans-splicing facilitates targeted delivery of suicide genes to cancer cells. Mol Cancer Ther 10: 233-41
-
(2011)
Mol Cancer Ther
, vol.10
, pp. 233-241
-
-
Gruber, C.1
Gratz, I.K.2
Murauer, E.M.3
-
17
-
-
70350622069
-
Is RNA manipulation a viable therapy for spinal muscular atrophy?
-
Horne C, Young PJ (2009) Is RNA manipulation a viable therapy for spinal muscular atrophy? J Neurol Sci 287:27-31
-
(2009)
J Neurol Sci
, vol.287
, pp. 27-31
-
-
Horne, C.1
Young, P.J.2
-
18
-
-
65649095625
-
Transsplicing as a novel method to rapidly produce antibody fusion proteins
-
Iwasaki R, Kiuchi H, Ihara M et al. (2009) Transsplicing as a novel method to rapidly produce antibody fusion proteins. Biochem Biophys Res Commun 384:316-21
-
(2009)
Biochem Biophys Res Commun
, vol.384
, pp. 316-321
-
-
Iwasaki, R.1
Kiuchi, H.2
Ihara, M.3
-
19
-
-
4444371896
-
Restoration of protein synthesis in pancreatic cancer cells by trans-splicing ribozymes
-
Kastanos E, Hjiantoniou E, Phylactou LA (2004) Restoration of protein synthesis in pancreatic cancer cells by trans-splicing ribozymes. Biochem Biophys Res Commun 322:930-4
-
(2004)
Biochem Biophys Res Commun
, vol.322
, pp. 930-934
-
-
Kastanos, E.1
Hjiantoniou, E.2
Phylactou, L.A.3
-
20
-
-
34447542621
-
Using 50-PTMs to repair mutant beta-globin transcripts
-
Kierlin-Duncan MN, Sullenger BA (2007) Using 50-PTMs to repair mutant beta-globin transcripts. RNA 13:1317-27
-
(2007)
RNA
, vol.13
, pp. 1317-1327
-
-
Kierlin-Duncan, M.N.1
Sullenger, B.A.2
-
21
-
-
0034834346
-
Promiscuity of pre-mRNA spliceosome-mediated trans splicing: A problem for gene therapy?
-
Kikumori T, Cote GJ, Gagel RF (2001) Promiscuity of pre-mRNA spliceosome-mediated trans splicing: a problem for gene therapy? Hum Gene Ther 12:1429-41
-
(2001)
Hum Gene Ther
, vol.12
, pp. 1429-1441
-
-
Kikumori, T.1
Cote, G.J.2
Gagel, R.F.3
-
22
-
-
85059107382
-
Improvement of RNA trans-splicing based gene correction using a RTM screening system
-
(abstr.)
-
Koller U, Wally V, Mitchell LG et al. (2010) Improvement of RNA trans-splicing based gene correction using a RTM screening system. Hum Gene Ther 21:S1451 (abstr.)
-
(2010)
Hum Gene Ther
, vol.21
-
-
Koller, U.1
Wally, V.2
Mitchell, L.G.3
-
23
-
-
80052498191
-
A novel screening system improves genetic correction by internal exon replacement
-
Koller U, Wally V, Mitchell LG et al. (2011) A novel screening system improves genetic correction by internal exon replacement. Nuc Acids Res 39:e108
-
(2011)
Nuc Acids Res
, vol.39
-
-
Koller, U.1
Wally, V.2
Mitchell, L.G.3
-
24
-
-
24944551962
-
Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fibrosis transmembrane conductance regulator function to polarized human cystic fibrosis airway epithelial cells
-
Liu XM, Luo MH, Zhang LN et al. (2005) Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fibrosis transmembrane conductance regulator function to polarized human cystic fibrosis airway epithelial cells. Hum Gene Ther 16:1116-23
-
(2005)
Hum Gene Ther
, vol.16
, pp. 1116-1123
-
-
Liu, X.M.1
Luo, M.H.2
Zhang, L.N.3
-
25
-
-
77956509696
-
Exon exchange approach to repair duchenne dystrophin transcripts
-
Lorain S, Peccate C, Le Hir M et al. (2010) Exon exchange approach to repair duchenne dystrophin transcripts. Plos One 5:e10894
-
(2010)
Plos One
, vol.5
-
-
Lorain, S.1
Peccate, C.2
Le Hir, M.3
-
26
-
-
0141743742
-
50 exon replacement and repair by spliceosome-mediated RNA trans-splicing
-
Mansfield SG, Clark RH, Puttaraju M et al. (2003) 50 exon replacement and repair by spliceosome-mediated RNA trans-splicing. RNA 9:1290-7
-
(2003)
RNA
, vol.9
, pp. 1290-1297
-
-
Mansfield, S.G.1
Clark, R.H.2
Puttaraju, M.3
-
27
-
-
33845524625
-
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells
-
Mavilio F, Pellegrini G, Ferrari S et al. (2006) Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nat Med 12:1397-402
-
(2006)
Nat Med
, vol.12
, pp. 1397-1402
-
-
Mavilio, F.1
Pellegrini, G.2
Ferrari, S.3
-
28
-
-
85059107716
-
A 50 trans-splicing gene therapy approach for dystrophic epidermolysis bullosa
-
(abstr.)
-
Mayr E, Koller U, Murauer EM et al. (2011) A 50 trans-splicing gene therapy approach for dystrophic epidermolysis bullosa. J Invest Dermatol 131:S68 (abstr.)
-
(2011)
J Invest Dermatol
, vol.131
-
-
Mayr, E.1
Koller, U.2
Murauer, E.M.3
-
29
-
-
27144477400
-
Gene therapy progress and prospects: Reprograming gene expression by trans-splicing
-
Mitchell LG, McGarrity GJ (2005) Gene therapy progress and prospects: reprograming gene expression by trans-splicing. Gene Ther 12:1477-85
-
(2005)
Gene Ther
, vol.12
, pp. 1477-1485
-
-
Mitchell, L.G.1
McGarrity, G.J.2
-
31
-
-
78650297492
-
Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa
-
Murauer EM, Gache Y, Gratz I et al. (2011) Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa. J Invest Dermatol 131:74-83
-
(2011)
J Invest Dermatol
, vol.131
, pp. 74-83
-
-
Murauer, E.M.1
Gache, Y.2
Gratz, I.3
-
32
-
-
84863986900
-
30 trans-splicing repair of COL7A1 mutations in recessive dystrophic epidermolysis bullosa patients
-
(abstr.)
-
Murauer EM, Gache Y, Larcher F et al. (2010) 30 trans-splicing repair of COL7A1 mutations in recessive dystrophic epidermolysis bullosa patients. J Invest Dermatol 130:S75 (abstr.)
-
(2010)
J Invest Dermatol
, vol.130
-
-
Murauer, E.M.1
Gache, Y.2
Larcher, F.3
-
33
-
-
0022975688
-
Identification of a novel y branch structure as an intermediate in trypanosome mRNA processing: Evidence for trans splicing
-
Murphy WJ, Watkins KP, Agabian N (1986) Identification of a novel Y branch structure as an intermediate in trypanosome mRNA processing: evidence for trans splicing. Cell 47:517-25
-
(1986)
Cell
, vol.47
, pp. 517-525
-
-
Murphy, W.J.1
Watkins, K.P.2
Agabian, N.3
-
34
-
-
11244318660
-
Gene transfer-mediated pre-mRNA segmental trans-splicing as a strategy to deliver intracellular toxins for cancer therapy
-
Nakayama K, Pergolizzi RG, Crystal RG (2005) Gene transfer-mediated pre-mRNA segmental trans-splicing as a strategy to deliver intracellular toxins for cancer therapy. Cancer Res 65:254-63
-
(2005)
Cancer Res
, vol.65
, pp. 254-263
-
-
Nakayama, K.1
Pergolizzi, R.G.2
Crystal, R.G.3
-
35
-
-
77952826869
-
Genetic correction of sickle cell anemia and beta-thalassemia: Progress and new perspective
-
Perumbeti A, Malik P (2010) Genetic correction of sickle cell anemia and beta-thalassemia: progress and new perspective. Scientific World Journal 10:644-54
-
(2010)
Scientific World Journal
, vol.10
, pp. 644-654
-
-
Perumbeti, A.1
Malik, P.2
-
36
-
-
0034889309
-
Messenger RNA repair and restoration of protein function by spliceosomemediated RNA trans-splicing
-
Puttaraju M, DiPasquale J, Baker CC et al. (2001) Messenger RNA repair and restoration of protein function by spliceosomemediated RNA trans-splicing. Mol Ther 4: 105-14
-
(2001)
Mol Ther
, vol.4
, pp. 105-114
-
-
Puttaraju, M.1
Dipasquale, J.2
Baker, C.C.3
-
37
-
-
0032993842
-
Spliceosome-mediated RNA transsplicing as a tool for gene therapy
-
Puttaraju M, Jamison SF, Mansfield SG et al. (1999) Spliceosome-mediated RNA transsplicing as a tool for gene therapy. Nat Biotechnol 17:246-52
-
(1999)
Nat Biotechnol
, vol.17
, pp. 246-252
-
-
Puttaraju, M.1
Jamison, S.F.2
Mansfield, S.G.3
-
38
-
-
68749092850
-
Correction of tau missplicing caused by FTDP-17 MAPT mutations by spliceosome-mediated RNA transsplicing
-
Rodriguez-Martin T, Anthony K, Garcia-Blanco MA et al. (2009) Correction of tau missplicing caused by FTDP-17 MAPT mutations by spliceosome-mediated RNA transsplicing. Hum Mol Genet 18:3266-73
-
(2009)
Hum Mol Genet
, vol.18
, pp. 3266-3273
-
-
Rodriguez-Martin, T.1
Anthony, K.2
Garcia-Blanco, M.A.3
-
39
-
-
27344453374
-
Reprogramming of tau alternative splicing by spliceosomemediated RNA trans-splicing: Implications for tauopathies
-
Rodriguez-Martin T, Garcia-Blanco MA, Mansfield SG et al. (2005) Reprogramming of tau alternative splicing by spliceosomemediated RNA trans-splicing: implications for tauopathies. Proc Natl Acad Sci USA 102:15659-64
-
(2005)
Proc Natl Acad Sci USA
, vol.102
, pp. 15659-15664
-
-
Rodriguez-Martin, T.1
Garcia-Blanco, M.A.2
Mansfield, S.G.3
-
40
-
-
0242321277
-
In-cell generation of antibody single-chain Fv transcripts by targeted RNA trans-splicing
-
Schlesinger J, Arama D, Noy H et al. (2003) In-cell generation of antibody single-chain Fv transcripts by targeted RNA trans-splicing. J Immunol Meth 282:175-86
-
(2003)
J Immunol Meth
, vol.282
, pp. 175-186
-
-
Schlesinger, J.1
Arama, D.2
Noy, H.3
-
41
-
-
79951826869
-
Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy
-
Shababi M, Glascock J, Lorson CL (2011) 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: 135-44
-
(2011)
Hum Gene Ther
, vol.22
, pp. 135-144
-
-
Shababi, M.1
Glascock, J.2
Lorson, C.L.3
-
42
-
-
84859158576
-
Optimization of SMN trans-splicing through the analysis of SMN introns
-
Shababi M, Lorson CL (2011) Optimization of SMN trans-splicing through the analysis of SMN introns. J Mol Neurosci 46:459-69
-
(2011)
J Mol Neurosci
, vol.46
, pp. 459-469
-
-
Shababi, M.1
Lorson, C.L.2
-
43
-
-
4344614627
-
Ribozymemediated induction of apoptosis in human cancer cells by targeted repair of mutant p53 RNA
-
Shin KS, Sullenger BA, Lee SW (2004) Ribozymemediated induction of apoptosis in human cancer cells by targeted repair of mutant p53 RNA. Mol Ther 10:365-72
-
(2004)
Mol Ther
, vol.10
, pp. 365-372
-
-
Shin, K.S.1
Sullenger, B.A.2
Lee, S.W.3
-
44
-
-
58749096360
-
On the road to reading the RNA-interference code
-
Siomi H, Siomi MC (2009) On the road to reading the RNA-interference code. Nature 457: 396-404
-
(2009)
Nature
, vol.457
, pp. 396-404
-
-
Siomi, H.1
Siomi, M.C.2
-
45
-
-
62549141507
-
Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing
-
Song Y, Lou HH, Boyer JL et al. (2009) Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing. Hum Gene Ther 20:267-81
-
(2009)
Hum Gene Ther
, vol.20
, pp. 267-281
-
-
Song, Y.1
Lou, H.H.2
Boyer, J.L.3
-
46
-
-
0027987902
-
Ribozymemediated repair of defective mRNA by targeted, trans-splicing
-
Sullenger BA, Cech TR (1994) Ribozymemediated repair of defective mRNA by targeted, trans-splicing. Nature 371:619-22
-
(1994)
Nature
, vol.371
, pp. 619-622
-
-
Sullenger, B.A.1
Cech, T.R.2
-
47
-
-
4043179784
-
Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency
-
Tahara M, Pergolizzi RG, Kobayashi H et al. (2004) Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency. Nat Med 10: 835-41
-
(2004)
Nat Med
, vol.10
, pp. 835-841
-
-
Tahara, M.1
Pergolizzi, R.G.2
Kobayashi, H.3
-
48
-
-
46749148868
-
A generalizable strategy for imaging premRNA levels in living subjects using spliceosome-mediated RNA trans-splicing
-
Walls ZF, Puttaraju M, Temple GF et al. (2008) A generalizable strategy for imaging premRNA levels in living subjects using spliceosome-mediated RNA trans-splicing. J Nucl Med 49:1146-54
-
(2008)
J Nucl Med
, vol.49
, pp. 1146-1154
-
-
Walls, Z.F.1
Puttaraju, M.2
Temple, G.F.3
-
49
-
-
78149237578
-
K14 mRNA reprogramming for dominant epidermolysis bullosa simplex
-
Wally V, Brunner M, Lettner T et al. (2010) K14 mRNA reprogramming for dominant epidermolysis bullosa simplex. Hum Mol Genet 19:4715-25
-
(2010)
Hum Mol Genet
, vol.19
, pp. 4715-4725
-
-
Wally, V.1
Brunner, M.2
Lettner, T.3
-
51
-
-
84878299265
-
Highthroughput screening for highly functional RNA-trans-splicing molecules: Correction of plectin in epidermolysis bullosa simplex
-
Plaseska-Karanfilska D (ed.) InTech Open Access Publisher: Rijeka
-
Wally V, Koller U, Bauer JW (2011) Highthroughput screening for highly functional RNA-trans-splicing molecules: correction of plectin in epidermolysis bullosa simplex. In: Plaseska-Karanfilska D (ed.) Genetic Disease. InTech Open Access Publisher: Rijeka
-
(2011)
Genetic Disease
-
-
Wally, V.1
Koller, U.2
Bauer, J.W.3
-
52
-
-
63649133449
-
Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivo
-
Wang J, Mansfield SG, Cote CA et al. (2009) Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivo. Mol Ther 17:343-51
-
(2009)
Mol Ther
, vol.17
, pp. 343-351
-
-
Wang, J.1
Mansfield, S.G.2
Cote, C.A.3
-
53
-
-
1542283819
-
Epidermolysis bullosa simplex-type mutations alter the dynamics of the keratin cytoskeleton and reveal a contribution of actin to the transport of keratin subunits
-
Werner NS, Windorffer R, Sternad P et al. (2004) Epidermolysis bullosa simplex-type mutations alter the dynamics of the keratin cytoskeleton and reveal a contribution of actin to the transport of keratin subunits. Mol Biol Cell 15:990-1002
-
(2004)
Mol Biol Cell
, vol.15
, pp. 990-1002
-
-
Werner, N.S.1
Windorffer, R.2
Sternad, P.3
-
54
-
-
28444453994
-
Spliceosome-mediated RNA trans-splicing
-
Yang YP, Walsh CE (2005) Spliceosome-mediated RNA trans-splicing. Mol Ther 12:1006-12
-
(2005)
Mol Ther
, vol.12
, pp. 1006-1012
-
-
Yang, Y.P.1
Walsh, C.E.2
-
55
-
-
34250739824
-
Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery
-
Zayed H, Xia L, Yerich A et al. (2007) Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery. Mol Ther 15:1273-9
-
(2007)
Mol Ther
, vol.15
, pp. 1273-1279
-
-
Zayed, H.1
Xia, L.2
Yerich, A.3
|