-
1
-
-
4644361590
-
Inducible mouse models for inherited skin diseases: Implications for skin gene therapy
-
Arin MJ, Roop DR (2004) Inducible mouse models for inherited skin diseases: implications for skin gene therapy. Cells Tissues Organs 177:160-8
-
(2004)
Cells Tissues Organs
, vol.177
, pp. 160-168
-
-
Arin, M.J.1
Roop, D.R.2
-
2
-
-
0042388694
-
Phenotype correction of hemophilia A in mice by spliceosome-mediated RNA trans-splicing
-
Chao H, Mansfield SG, Bartel RC, Hiriyana S, Mitchell LG, Garcia-Blanco MA et al. (2003) Phenotype correction of hemophilia A in mice by spliceosome-mediated RNA trans-splicing. Nat Med 9:1015-9
-
(2003)
Nat Med
, vol.9
, pp. 1015-1019
-
-
Chao, H.1
Mansfield, S.G.2
Bartel, R.C.3
Hiriyana, S.4
Mitchell, L.G.5
Garcia-Blanco, M.A.6
-
3
-
-
0142084745
-
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
-
Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P et al. (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302:415-9
-
(2003)
Science
, vol.302
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
McCormack, M.P.4
Wulffraat, N.5
Leboulch, P.6
-
4
-
-
33750616005
-
Gene therapy progress and prospects: The skin - easily accessible but still far away
-
Hengge UR (2006) Gene therapy progress and prospects: the skin - easily accessible but still far away. Gene Ther 13:1555-63
-
(2006)
Gene Ther
, vol.13
, pp. 1555-1563
-
-
Hengge, U.R.1
-
5
-
-
0036137663
-
Partial correction of endogenous deltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing
-
Liu X, Jiang Q, Mansfield SG, Puttaraju M, Zhang Y, Zhou W et al. (2002) Partial correction of endogenous deltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing. Nat Biotechnol 20:47-52
-
(2002)
Nat Biotechnol
, vol.20
, pp. 47-52
-
-
Liu, X.1
Jiang, Q.2
Mansfield, S.G.3
Puttaraju, M.4
Zhang, Y.5
Zhou, W.6
-
6
-
-
0141743742
-
5 exon replacement and repair by spliceosome-mediated RNA trans-splicing
-
Mansfield SG, Clark RH, Puttaraju M, Kole J, Cohn JA, Mitchell LG et al. (2003) 5 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
Kole, J.4
Cohn, J.A.5
Mitchell, L.G.6
-
7
-
-
0037244019
-
Injection of genetically engineered fibroblasts corrects regenerated human epidermolysis bullosa skin tissue
-
Ortiz-Urda S, Lin Q, Green CL, Keene DR, Marinkovich MP, Khavari PA (2003a) Injection of genetically engineered fibroblasts corrects regenerated human epidermolysis bullosa skin tissue. J Clin Invest 111:251-5
-
(2003)
J Clin Invest
, vol.111
, pp. 251-255
-
-
Ortiz-Urda, S.1
Lin, Q.2
Green, C.L.3
Keene, D.R.4
Marinkovich, M.P.5
Khavari, P.A.6
-
8
-
-
0038618975
-
PhiC31 integrase mediated nonviral genetic correction of junctional epidermolysis bullosa
-
Ortiz-Urda S, Thyagarajan B, Keene DR, Lin Q, Calos MP, Khavari PA (2003b) PhiC31 integrase mediated nonviral genetic correction of junctional epidermolysis bullosa. Hum Gene Ther 14:923-8
-
(2003)
Hum Gene Ther
, vol.14
, pp. 923-928
-
-
Ortiz-Urda, S.1
Thyagarajan, B.2
Keene, D.R.3
Lin, Q.4
Calos, M.P.5
Khavari, P.A.6
-
9
-
-
7444248879
-
Progress in epidermolysis bullosa: Genetic classification and clinical implications
-
Uitto J, Richard G (2004) Progress in epidermolysis bullosa: genetic classification and clinical implications. Am J Med Genet C 131:61-74
-
(2004)
Am J Med Genet C
, vol.131
, pp. 61-74
-
-
Uitto, J.1
Richard, G.2
-
10
-
-
18944373328
-
Highly efficient endogenous human gene correction using designed zinc-finger nucleases
-
Urnov FD, Miller JC, Lee YL, Beausejour CM, Rock JM, Augustus S et al. (2005) Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435:646-51
-
(2005)
Nature
, vol.435
, pp. 646-651
-
-
Urnov, F.D.1
Miller, J.C.2
Lee, Y.L.3
Beausejour, C.M.4
Rock, J.M.5
Augustus, S.6
-
11
-
-
39149136181
-
5′ Trans-splicing repair of the PLEC1 gene
-
Wally V, Klausegger A, Koller U, Lochmüller H, Krause S, Wiche G et al. (2008) 5′ Trans-splicing repair of the PLEC1 gene. J Invest Dermatol 128:568-74
-
(2008)
J Invest Dermatol
, vol.128
, pp. 568-574
-
-
Wally, V.1
Klausegger, A.2
Koller, U.3
Lochmüller, H.4
Krause, S.5
Wiche, G.6
|