-
1
-
-
0042388694
-
Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing
-
Chao, H.; Mansfield, S.G.; Bartel, R.C.; Hiriyanna, S.; Mitchell, L.G.; Garcia-Blanco, M.A.; Walsh, C.E. Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing. Nat. Med. 2003, 9, 1015-1019.
-
(2003)
Nat. Med
, vol.9
, pp. 1015-1019
-
-
Chao, H.1
Mansfield, S.G.2
Bartel, R.C.3
Hiriyanna, S.4
Mitchell, L.G.5
Garcia-Blanco, M.A.6
Walsh, C.E.7
-
2
-
-
0141743742
-
5' Exon replacement and repair by spliceosome-mediated RNA trans-splicing
-
Mansfield, S.G.; Clark, R.H.; Puttaraju, M.; Kole, J.; Cohn, J.A.; Mitchell, L.G.; Garcia-Blanco, M.A. 5' Exon replacement and repair by spliceosome-mediated RNA trans-splicing. RNA 2003, 9, 1290-1297.
-
(2003)
RNA
, vol.9
, pp. 1290-1297
-
-
Mansfield, S.G.1
Clark, R.H.2
Puttaraju, M.3
Kole, J.4
Cohn, J.A.5
Mitchell, L.G.6
Garcia-Blanco, M.A.7
-
3
-
-
39149136181
-
5' Trans-splicing repair of the PLEC1 gene
-
Wally, V.; Klausegger, A.; Koller, U.; Lochmuller, H.; Krause, S.; Wiche, G.; Mitchell, L.G.; Hintner, H.; Bauer, J.W. 5' Trans-splicing repair of the PLEC1 gene. J. Investig. Dermatol. 2008, 128, 568-574.
-
(2008)
J. Investig. Dermatol
, vol.128
, pp. 568-574
-
-
Wally, V.1
Klausegger, A.2
Koller, U.3
Lochmuller, H.4
Krause, S.5
Wiche, G.6
Mitchell, L.G.7
Hintner, H.8
Bauer, J.W.9
-
4
-
-
78149237578
-
K14 mRNA reprogramming for dominant epidermolysis bullosa simplex
-
Wally, V.; Brunner, M.; Lettner, T.; Wagner, M.; Koller, U.; Trost, A.; Murauer, E.M.; Hainzl, S.; Hintner, H.; Bauer, J.W. K14 mRNA reprogramming for dominant epidermolysis bullosa simplex. Hum. Mol. Genet. 2010, 19, 4715-4725.
-
(2010)
Hum. Mol. Genet
, vol.19
, pp. 4715-4725
-
-
Wally, V.1
Brunner, M.2
Lettner, T.3
Wagner, M.4
Koller, U.5
Trost, A.6
Murauer, E.M.7
Hainzl, S.8
Hintner, H.9
Bauer, J.W.10
-
5
-
-
78650297492
-
Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa
-
Murauer, E.M.; Gache, Y.; Gratz, I.K.; Klausegger, A.; Muss, W.; Gruber, C.; Meneguzzi, G.; Hintner, H.; Bauer, J.W. Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa. J. Investig. Dermatol. 2011, 131, 74-83.
-
(2011)
J. Investig. Dermatol
, vol.131
, pp. 74-83
-
-
Murauer, E.M.1
Gache, Y.2
Gratz, I.K.3
Klausegger, A.4
Muss, W.5
Gruber, C.6
Meneguzzi, G.7
Hintner, H.8
Bauer, J.W.9
-
6
-
-
84863983970
-
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
-
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
-
8
-
-
80052498191
-
A novel screening system improves genetic correction by internal exon replacement
-
Koller, U.; Wally, V.; Mitchell, L.G.; Klausegger, A.; Murauer, E.M.; Mayr, E.; Gruber, C.; Hainzl, S.; Hintner, H.; Bauer, J.W. A novel screening system improves genetic correction by internal exon replacement. Nucleic Acids Res. 2011, 39, e108.
-
(2011)
Nucleic Acids Res
, vol.39
-
-
Koller, U.1
Wally, V.2
Mitchell, L.G.3
Klausegger, A.4
Murauer, E.M.5
Mayr, E.6
Gruber, C.7
Hainzl, S.8
Hintner, H.9
Bauer, J.W.10
-
9
-
-
84878299265
-
-
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
-
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
-
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
-
12
-
-
84879304502
-
RNA trans-splicing for genodermatoses
-
Bauer, J.W.; Murauer, E.M.; Wally, V.; Koller, U. RNA trans-splicing for genodermatoses. Methods Mol. Biol. 2013, 961, 441-455.
-
(2013)
Methods Mol. Biol
, vol.961
, pp. 441-455
-
-
Bauer, J.W.1
Murauer, E.M.2
Wally, V.3
Koller, U.4
-
13
-
-
55649102490
-
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
-
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
-
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
-
17
-
-
84901242657
-
Inherited epidermolysis bullosa: Updated recommendations on diagnosis and classification
-
Fine, J.D.; Bruckner-Tuderman, L.; Eady, R.A.; Bauer, E.A.; Bauer, J.W.; Has, C.; Heagerty, A.; Hintner, H.; Hovnanian, A.; Jonkman, M.F., et al. Inherited epidermolysis bullosa: Updated recommendations on diagnosis and classification. J. Am. Acad. Dermatol. 2014, 70, 1103-1126.
-
(2014)
J. Am. Acad. Dermatol.
, vol.70
, pp. 1103-1126
-
-
Fine, J.D.1
Bruckner-Tuderman, L.2
Eady, R.A.3
Bauer, E.A.4
Bauer, J.W.5
Has, C.6
Heagerty, A.7
Hintner, H.8
Hovnanian, A.9
Jonkman, M.F.10
-
18
-
-
79951834142
-
Spliceosome-mediated RNA trans-splicing facilitates targeted delivery of suicide genes to cancer cells
-
Gruber, C.; Gratz, I.K.; Murauer, E.M.; Mayr, E.; Koller, U.; Bruckner-Tuderman, L.; Meneguzzi, G.; Hintner, H.; Bauer, J.W. Spliceosome-mediated RNA trans-splicing facilitates targeted delivery of suicide genes to cancer cells. Mol. Cancer Ther. 2011, 10, 233-241.
-
(2011)
Mol. Cancer Ther
, vol.10
, pp. 233-241
-
-
Gruber, C.1
Gratz, I.K.2
Murauer, E.M.3
Mayr, E.4
Koller, U.5
Bruckner-Tuderman, L.6
Meneguzzi, G.7
Hintner, H.8
Bauer, J.W.9
-
19
-
-
84887930665
-
The design and optimization of RNA trans-splicing molecules for skin cancer therapy
-
Gruber, C.; Koller, U.; Murauer, E.M.; Hainzl, S.; Hüttner, C.; Kocher, T.; South, A.P.; Hintner, H.; Bauer, J.W. The design and optimization of RNA trans-splicing molecules for skin cancer therapy. Mol. Oncol. 2013, 7, 1056-1068.
-
(2013)
Mol. Oncol
, vol.7
, pp. 1056-1068
-
-
Gruber, C.1
Koller, U.2
Murauer, E.M.3
Hainzl, S.4
Hüttner, C.5
Kocher, T.6
South, A.P.7
Hintner, H.8
Bauer, J.W.9
-
20
-
-
0032993842
-
Spliceosome-mediated RNA trans-splicing as a tool for gene therapy
-
Puttaraju, M.; Jamison, S.F.; Mansfield, S.G.; Garcia-Blanco, M.A.; Mitchell, L.G. Spliceosome-mediated RNA trans-splicing as a tool for gene therapy. Nat. Biotechnol. 1999, 17, 246-225.
-
(1999)
Nat. Biotechnol
, vol.17
, pp. 225-246
-
-
Puttaraju, M.1
Jamison, S.F.2
Mansfield, S.G.3
Garcia-Blanco, M.A.4
Mitchell, L.G.5
-
21
-
-
27144477400
-
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
-
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
-
23
-
-
43049093343
-
A hypomorphic mouse model of dystrophic epidermolysis bullosa reveals mechanisms of disease and response to fibroblast therapy
-
Fritsch, A.; Loeckermann, S.; Kern, J.S.; Braun, A.; Bosl, M.R.; Bley, T.A.; Schumann, H.; von Elverfeldt, D.; Paul, D.; Erlacher, M., et al. A hypomorphic mouse model of dystrophic epidermolysis bullosa reveals mechanisms of disease and response to fibroblast therapy. J. Clin. Investig. 2008, 118, 1669-1679.
-
(2008)
J. Clin. Investig.
, vol.118
, pp. 1669-1679
-
-
Fritsch, A.1
Loeckermann, S.2
Kern, J.S.3
Braun, A.4
Bosl, M.R.5
Bley, T.A.6
Schumann, H.7
Von Elverfeldt, D.8
Paul, D.9
Erlacher, M.10
-
24
-
-
71049190003
-
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
-
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
|