-
1
-
-
84872479464
-
Enzyme replacement therapy for lysosomal diseases: Lessons from 20 years of experience and remaining challenges
-
Desnick RJ, Schuchman EH (2012) Enzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges. Ann Rev Genom Hum Genet 13:307-35
-
(2012)
Ann Rev Genom Hum Genet
, vol.13
, pp. 307-335
-
-
Desnick, R.J.1
Schuchman, E.H.2
-
2
-
-
43449084027
-
The classification of inherited epidermolysis bullosa (EB): Report of the Third International Consensus Meeting on Diagnosis and Classification of EB
-
Fine JD, Eady RA, Bauer EA et al. (2008) The classification of inherited epidermolysis bullosa (EB): Report of the Third International Consensus Meeting on Diagnosis and Classification of EB. J Am Acad Dermatol 58: 931-50
-
(2008)
J Am Acad Dermatol
, vol.58
, pp. 931-950
-
-
Fine, J.D.1
Eady, R.A.2
Bauer, E.A.3
-
3
-
-
43749107571
-
Protein therapeutics for junctional epidermolysis bullosa: Incorporation of recombinant β3 chain into laminin 332 in β3-/- keratinocytes in vitro
-
DOI 10.1038/sj.jid.5701197, PII 5701197
-
Igoucheva O, Kelly A, Uitto J et al. (2008) Protein therapeutics for junctional epidermolysis bullosa: incorporation of recombinant beta3 chain into laminin 332 in beta3 -/- keratino-cytes in vitro. J Invest Dermatol 128:1476-86 (Pubitemid 351693189)
-
(2008)
Journal of Investigative Dermatology
, vol.128
, Issue.6
, pp. 1476-1486
-
-
Igoucheva, O.1
Kelly, A.2
Uitto, J.3
Alexeev, V.4
-
4
-
-
33845524625
-
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells
-
DOI 10.1038/nm1504, PII NM1504
-
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 (Pubitemid 44924498)
-
(2006)
Nature Medicine
, vol.12
, Issue.12
, pp. 1397-1402
-
-
Mavilio, F.1
Pellegrini, G.2
Ferrari, S.3
Di Nunzio, F.4
Di Iorio, E.5
Recchia, A.6
Maruggi, G.7
Ferrari, G.8
Provasi, E.9
Bonini, C.10
Capurro, S.11
Conti, A.12
Magnoni, C.13
Giannetti, A.14
De Luca, M.15
-
5
-
-
77954541601
-
Immune reactivity to type VII collagen: Implications for gene therapy of recessive dystrophic epidermolysis bullosa
-
Pendaries V, Gasc G, Titeux M et al. (2010) Immune reactivity to type VII collagen: implications for gene therapy of recessive dystrophic epidermolysis bullosa. Gene Ther 17:930-7
-
(2010)
Gene Ther
, vol.17
, pp. 930-937
-
-
Pendaries, V.1
Gasc, G.2
Titeux, M.3
-
6
-
-
58149250413
-
Injection of recombinant human type VII collagen corrects the disease phenotype in a murine model of dystrophic epidermolysis bullosa
-
Remington J, Wang X, Hou Y et al. (2009) Injection of recombinant human type VII collagen corrects the disease phenotype in a murine model of dystrophic epidermolysis bullosa. Mol Ther 17:26-33
-
(2009)
Mol Ther
, vol.17
, pp. 26-33
-
-
Remington, J.1
Wang, X.2
Hou, Y.3
-
7
-
-
77958109358
-
Long-term type VII collagen restoration to human epidermolysis bullosa skin tissue
-
Siprashvili Z, Nguyen NT, Bezchinsky MY et al. (2010) Long-term type VII collagen restoration to human epidermolysis bullosa skin tissue. Hum Gene Ther21:1299-310
-
(2010)
Hum Gene Ther
, vol.21
, pp. 1299-1310
-
-
Siprashvili, Z.1
Nguyen, N.T.2
Bezchinsky, M.Y.3
-
8
-
-
77955171264
-
SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa
-
Titeux M, Pendaries V, Zanta-Boussif MA et al. (2010) SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa. Mol Ther 18:1509-18
-
(2010)
Mol Ther
, vol.18
, pp. 1509-1518
-
-
Titeux, M.1
Pendaries, V.2
Zanta-Boussif, M.A.3
-
9
-
-
77956621310
-
Bone marrow transplantation for recessive dystrophic epidermolysis bullosa
-
Wagner JE, Ishida-Yamamoto A, JMcGrath JA et al. (2010) Bone marrow transplantation for recessive dystrophic epidermolysis bullosa. New Engl J Med 363:629-3 9
-
(2010)
New Engl J Med
, vol.363
, pp. 629-639
-
-
Wagner, J.E.1
Ishida-Yamamoto, A.2
Jmcgrath, J.A.3
-
10
-
-
49549096936
-
Potential of fibroblast cell therapy for recessive dystrophic epidermolysis bullosa
-
Wong T, Gammon L, Liu L et al. (2008) Potential of fibroblast cell therapy for recessive dystrophic epidermolysis bullosa. J Invest Dermatol 128:2179-89
-
(2008)
J Invest Dermatol
, vol.128
, pp. 2179-2189
-
-
Wong, T.1
Gammon, L.2
Liu, L.3
-
11
-
-
3142713644
-
Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa
-
DOI 10.1038/nm1063
-
Woodley DT, Keene DR, Atha T et al. (2004) Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa. Nat Med 10:693-5 (Pubitemid 38937633)
-
(2004)
Nature Medicine
, vol.10
, Issue.7
, pp. 693-695
-
-
Woodley, D.T.1
Keene, D.R.2
Atha, T.3
Huang, Y.4
Lipman, K.5
Li, W.6
Chen, M.7
-
12
-
-
33847182092
-
Intravenously injected human fibroblasts home to skin wounds, deliver type VII collagen, and promote wound healing
-
Woodley DT, Remington J, Huang Y et al. (2007) Intravenously injected human fibroblasts home to skin wounds, deliver type VII collagen, and promote wound healing. Mol Ther 15:628-35
-
(2007)
Mol Ther
, vol.15
, pp. 628-635
-
-
Woodley, D.T.1
Remington, J.2
Huang, Y.3
-
13
-
-
84879412500
-
Intravenously injected recombinant human type VII collagen homes to skin wounds and restrores skin intergrity of dystrophic epidermolysis bullosa
-
Woodley DT, Wang X, Amir M et al. (2013) Intravenously injected recombinant human type VII collagen homes to skin wounds and restrores skin intergrity of dystrophic epidermolysis bullosa. J Invest Dermatol 133:1910-3.
-
(2013)
J Invest Dermatol
, vol.133
, pp. 1910-1913
-
-
Woodley, D.T.1
Wang, X.2
Amir, M.3
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