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Volumn 24, Issue 4, 2013, Pages 182-190

Phase i study protocol for Ex vivo lentiviral gene therapy for the inherited skin disease, netherton syndrome

Author keywords

[No Author keywords available]

Indexed keywords

DERMATOLOGICAL AGENT; IMMUNOGLOBULIN E; LENTIVIRUS VECTOR; LYMPHOEPITHELIAL KAZAL TYPE RELATED INHIBITOR; SERINE PROTEINASE INHIBITOR; UNCLASSIFIED DRUG;

EID: 84907241768     PISSN: 23248637     EISSN: 23248645     Source Type: Journal    
DOI: 10.1089/humc.2013.195     Document Type: Article
Times cited : (33)

References (37)
  • 1
    • 84879867061 scopus 로고    scopus 로고
    • Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
    • Aiuti, A., et al. (2013). Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 341, 1233151.
    • (2013) Science , vol.341 , pp. 1233151
    • Aiuti, A.1
  • 2
    • 84879873039 scopus 로고    scopus 로고
    • Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
    • Biffi, A., et al. (2013). Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341, 1233158.
    • (2013) Science , vol.341 , pp. 1233158
    • Biffi, A.1
  • 4
    • 77949752021 scopus 로고    scopus 로고
    • Elastase 2 is expressed in human and mouse epidermis and impairs skin barrier function in Neth-erton syndrome through filaggrin and lipid misprocessing
    • Bonnart, C., et al. (2010). Elastase 2 is expressed in human and mouse epidermis and impairs skin barrier function in Neth-erton syndrome through filaggrin and lipid misprocessing. J. Clin. Invest. 120, 871-882.
    • (2010) J. Clin. Invest. , vol.120 , pp. 871-882
    • Bonnart, C.1
  • 5
    • 66049125101 scopus 로고    scopus 로고
    • Kallikrein 5 induces atopic dermatitis-like lesions through PAR2-mediated thymic stromal lymphopoie-tin expression in Netherton syndrome
    • Briot, A., et al. (2009). Kallikrein 5 induces atopic dermatitis-like lesions through PAR2-mediated thymic stromal lymphopoie-tin expression in Netherton syndrome. J. Exp. Med. 206, 1135-1147.
    • (2009) J. Exp. Med. , vol.206 , pp. 1135-1147
    • Briot, A.1
  • 6
    • 84866652391 scopus 로고    scopus 로고
    • Gene therapy of skin adhesion disorders (mini review)
    • Cavazza, A., and Mavilio, F. (2012). Gene therapy of skin adhesion disorders (mini review). Curr. Pharm. Biotechnol. 13, 1868-1876.
    • (2012) Curr. Pharm. Biotechnol. , vol.13 , pp. 1868-1876
    • Cavazza, A.1    Mavilio, F.2
  • 7
    • 77956928344 scopus 로고    scopus 로고
    • Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia
    • Cavazzana-Calvo, M., et al. (2010). Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 467, 318-322.
    • (2010) Nature , vol.467 , pp. 318-322
    • Cavazzana-Calvo, M.1
  • 9
    • 0029906097 scopus 로고    scopus 로고
    • Corrective gene transfer in the human skin disorder lamellar ichthyosis
    • DOI 10.1038/nm1196-1263
    • Choate, K.A., Medalie, D.A., Morgan, J.R., and Khavari, P.A. (1996). Corrective gene transfer in the human skin disorder lamellar ichthyosis. Nat. Med. 2, 1263-1267. (Pubitemid 26375415)
    • (1996) Nature Medicine , vol.2 , Issue.11 , pp. 1263-1267
    • Choate, K.A.1    Medalie, D.A.2    Morgan, J.R.3    Khavari, P.A.4
  • 11
    • 85028120068 scopus 로고    scopus 로고
    • Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome-derived skin grafts
    • Di, W.L., et al. (2011). Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of Netherton syndrome-derived skin grafts. Mol. Ther. 19, 408-416.
    • (2011) Mol. Ther. , vol.19 , pp. 408-416
    • Di, W.L.1
  • 12
    • 84855867684 scopus 로고    scopus 로고
    • Human involucrin promoter mediates repression-resistant and compartment-specific LEKTI expression
    • Di, W.L., et al. (2012). Human involucrin promoter mediates repression-resistant and compartment-specific LEKTI expression. Hum. Gene Ther. 23, 83-90.
    • (2012) Hum. Gene Ther. , vol.23 , pp. 83-90
    • Di, W.L.1
  • 14
    • 0029918009 scopus 로고    scopus 로고
    • Loss of expression of a retrovirus-transduced gene in human keratinocytes
    • Fenjves, E.S., Yao, S.N., Kurachi, K., and Taichman, L.B. (1996). Loss of expression of a retrovirus-transduced gene in human keratinocytes. J. Invest. Dermatol. 106, 576-578.
    • (1996) J. Invest. Dermatol. , vol.106 , pp. 576-578
    • Fenjves, E.S.1    Yao, S.N.2    Kurachi, K.3    Taichman, L.B.4
  • 16
    • 0027465539 scopus 로고
    • Towards gene therapy for haemophilia B using primary human keratinocytes
    • DOI 10.1038/ng0293-180
    • Gerrard, A.J., Hudson, D.L., Brownlee, G.G., and Watt, F.M. (1993). Towards gene therapy for haemophilia B using primary human keratinocytes. Nat. Genet. 3, 180-183. (Pubitemid 23079803)
    • (1993) Nature Genetics , vol.3 , Issue.2 , pp. 180-183
    • Gerrard, A.J.1    Hudson, D.L.2    Brownlee, G.G.3    Watt, F.M.4
  • 17
    • 0036790465 scopus 로고    scopus 로고
    • Durable and stratum-specific gene expression in epidermis
    • Ghazizadeh, S., Doumeng, C., and Taichman, L.B. (2002). Durable and stratum-specific gene expression in epidermis. Gene Ther. 9, 1278-1285.
    • (2002) Gene Ther. , vol.9 , pp. 1278-1285
    • Ghazizadeh, S.1    Doumeng, C.2    Taichman, L.B.3
  • 19
    • 51349090473 scopus 로고    scopus 로고
    • Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
    • Hacein-Bey-Abina, S., et al. (2008). Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 118, 3132-3142.
    • (2008) J. Clin. Invest. , vol.118 , pp. 3132-3142
    • Hacein-Bey-Abina, S.1
  • 20
    • 33847330241 scopus 로고    scopus 로고
    • Clinical experience using cultured epithelial autografts leads to an alternative methodology for transferring skin cells from the laboratory to the patient
    • Hernon, C.A., et al. (2006). Clinical experience using cultured epithelial autografts leads to an alternative methodology for transferring skin cells from the laboratory to the patient. Regen. Med. 1, 809-821.
    • (2006) Regen. Med. , vol.1 , pp. 809-821
    • Hernon, C.A.1
  • 21
    • 51349158298 scopus 로고    scopus 로고
    • Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
    • Howe, S.J., et al. (2008). Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J. Clin. Invest. 118, 3143-3150.
    • (2008) J. Clin. Invest. , vol.118 , pp. 3143-3150
    • Howe, S.J.1
  • 23
    • 0036125197 scopus 로고    scopus 로고
    • Elevated stratum corneum hydrolytic activity in Netherton syndrome suggests an inhibitory regulation of desquamation by SPINK5-derived peptides
    • DOI 10.1046/j.0022-202x.2001.01663.x
    • Komatsu, N., et al. (2002). Elevated stratum corneum hydrolytic activity in Netherton syndrome suggests an inhibitory regulation of desquamation by SPINK5-derived peptides. J. Invest. Dermatol. 118, 436-443. (Pubitemid 34227160)
    • (2002) Journal of Investigative Dermatology , vol.118 , Issue.3 , pp. 436-443
    • Komatsu, N.1    Takata, M.2    Otsuki, N.3    Ohka, R.4    Amano, O.5    Takehara, K.6    Saijoh, K.7
  • 25
    • 33847256045 scopus 로고    scopus 로고
    • Progress and opportunities for tissue-engineered skin
    • MacNeil, S. (2007). Progress and opportunities for tissue-engineered skin. Nature 445, 874-880.
    • (2007) Nature , vol.445 , pp. 874-880
    • Macneil, S.1
  • 26
    • 0039702803 scopus 로고    scopus 로고
    • LEKTI, a novel 15-domain type of human serine proteinase inhibitor
    • Magert, HJ., et al. (1999). LEKTI, a novel 15-domain type of human serine proteinase inhibitor. J. Biol. Chem. 274, 21499-21502.
    • (1999) J. Biol. Chem. , vol.274 , pp. 21499-21502
    • Magert, H.J.1
  • 27
    • 33750337565 scopus 로고    scopus 로고
    • Functional lentiviral vectors for xeroderma pigmentosum gene therapy
    • DOI 10.1016/j.jbiotec.2006.05.016, PII S0168165606004706
    • Marchetto, M.C., Correa, R.G., Menck, C.F., and Muotri, A.R. (2006). Functional lentiviral vectors for xeroderma pigmento-sum gene therapy. J. Biotechnol. 126, 424-430. (Pubitemid 44634821)
    • (2006) Journal of Biotechnology , vol.126 , Issue.4 , pp. 424-430
    • Marchetto, M.C.N.1    Correa, R.G.2    Menck, C.F.M.3    Muotri, A.R.4
  • 33
    • 33645734405 scopus 로고    scopus 로고
    • Correction of X-linked chronic granulo-matous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
    • Ott, M.G., et al. (2006). Correction of X-linked chronic granulo-matous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 12, 401-409.
    • (2006) Nat. Med. , vol.12 , pp. 401-409
    • Ott, M.G.1
  • 34
    • 0001951363 scopus 로고
    • Methods for clonal growth and serial cultuvation of normal human epidermal keratinocytes and mesothelial cells
    • R. Baserga, ed. (IRL Press, Oxford)
    • Rheinwald, J.G. (1989). Methods for clonal growth and serial cultuvation of normal human epidermal keratinocytes and mesothelial cells. In A Practical Approach. R. Baserga, ed. (IRL Press, Oxford), pp. 81-94.
    • (1989) A Practical Approach , pp. 81-94
    • Rheinwald, J.G.1
  • 35
    • 77955086483 scopus 로고    scopus 로고
    • Matriptase initiates activation of epidermal pro-kallikrein and disease onset in a mouse model of Netherton syndrome
    • Sales, K.U., et al. (2010). Matriptase initiates activation of epidermal pro-kallikrein and disease onset in a mouse model of Netherton syndrome. Nat. Genet. 42, 676-683.
    • (2010) Nat. Genet. , vol.42 , pp. 676-683
    • Sales, K.U.1
  • 37
    • 0036127719 scopus 로고    scopus 로고
    • The stem cell compartment in human interfollicular epidermis
    • DOI 10.1016/S0923-1811(02)00003-8, PII S0923181102000038
    • Watt, F.M. (2002). The stem cell compartment in human inter-follicular epidermis. J. Dermatol. Sci. 28, 173-180. (Pubitemid 34242651)
    • (2002) Journal of Dermatological Science , vol.28 , Issue.3 , pp. 173-180
    • Watt, F.M.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.