메뉴 건너뛰기




Volumn 132, Issue , 2012, Pages E29-E34

Molecular Therapeutics for Heritable Skin Diseases

Author keywords

[No Author keywords available]

Indexed keywords

GENE THERAPY; GENETIC DISORDER; GENETICS; HUMAN; METHODOLOGY; REVIEW; SKIN DISEASE; STEM CELL TRANSPLANTATION;

EID: 84879690707     PISSN: 0022202X     EISSN: 15231747     Source Type: Journal    
DOI: 10.1038/skinbio.2012.9     Document Type: Article
Times cited : (10)

References (48)
  • 1
    • 77949424341 scopus 로고    scopus 로고
    • Telomere elongation in induced pluripotent stem cells from dyskeratosis congenita patients
    • Agarwal, S., Loh, Y.H., McLoughlin, E.M., et al. Telomere elongation in induced pluripotent stem cells from dyskeratosis congenita patients. Nature 464 (2010), 292–296.
    • (2010) Nature , vol.464 , pp. 292-296
    • Agarwal, S.1    Loh, Y.H.2    McLoughlin, E.M.3
  • 2
    • 0038798707 scopus 로고    scopus 로고
    • Participation of bone marrow derived cells in cutaneous wound healing
    • Badiavas, E.V., Abedi, M., Butmarc, J., et al. Participation of bone marrow derived cells in cutaneous wound healing. J Cell Physiol 196 (2003), 245–250.
    • (2003) J Cell Physiol , vol.196 , pp. 245-250
    • Badiavas, E.V.1    Abedi, M.2    Butmarc, J.3
  • 3
    • 84872894722 scopus 로고    scopus 로고
    • Molecular heterogeneity of blistering disorders: the paradigm of epidermolysis bullosa
    • Bruckner-Tuderman, L., Has, C., Molecular heterogeneity of blistering disorders: the paradigm of epidermolysis bullosa. J Invest Dermatol 132 (2012), E2–E5.
    • (2012) J Invest Dermatol , vol.132 , pp. E2-E5
    • Bruckner-Tuderman, L.1    Has, C.2
  • 5
    • 0028011836 scopus 로고
    • Type VII collagen gene expression by human skin fibroblasts and keratinocytes in culture: influence of donor age and cytokine responses
    • Chen, Y.Q., Mauviel, A., Ryynanen, J., et al. Type VII collagen gene expression by human skin fibroblasts and keratinocytes in culture: influence of donor age and cytokine responses. J Invest Dermatol 102 (1994), 205–209.
    • (1994) J Invest Dermatol , vol.102 , pp. 205-209
    • Chen, Y.Q.1    Mauviel, A.2    Ryynanen, J.3
  • 6
    • 51349152151 scopus 로고    scopus 로고
    • Bone marrow cell transfer into fetal circulation can ameliorate genetic skin diseases by providing fibroblasts to the skin and inducing immune tolerance
    • Chino, T., Tamai, K., Yamazaki, T., et al. Bone marrow cell transfer into fetal circulation can ameliorate genetic skin diseases by providing fibroblasts to the skin and inducing immune tolerance. Am J Pathol 173 (2008), 803–814.
    • (2008) Am J Pathol , vol.173 , pp. 803-814
    • Chino, T.1    Tamai, K.2    Yamazaki, T.3
  • 7
    • 77950577869 scopus 로고    scopus 로고
    • Replenishment of type VII collagen and re-epithelialization of chronically ulcerated skin after intradermal administration of allogeneic mesenchymal stromal cells in two patients with recessive dystrophic epidermolysis bullosa
    • Conget, P., Rodriguez, F., Kramer, S., et al. Replenishment of type VII collagen and re-epithelialization of chronically ulcerated skin after intradermal administration of allogeneic mesenchymal stromal cells in two patients with recessive dystrophic epidermolysis bullosa. Cytotherapy 12 (2010), 429–431.
    • (2010) Cytotherapy , vol.12 , pp. 429-431
    • Conget, P.1    Rodriguez, F.2    Kramer, S.3
  • 8
    • 67649948869 scopus 로고    scopus 로고
    • Gene therapy of inherited skin adhesion disorders: a critical overview
    • De Luca, M., Pellegrini, G., Mavilio, F., Gene therapy of inherited skin adhesion disorders: a critical overview. Br J Dermatol 161 (2009), 19–24.
    • (2009) Br J Dermatol , vol.161 , pp. 19-24
    • De Luca, M.1    Pellegrini, G.2    Mavilio, F.3
  • 9
    • 70350068764 scopus 로고    scopus 로고
    • Phenotypic and genotypic analyses of genetic skin disease through the Online Mendelian Inheritance in Man (OMIM) database
    • Feramisco, J.D., Sadreyev, R.I., Murray, M.L., et al. Phenotypic and genotypic analyses of genetic skin disease through the Online Mendelian Inheritance in Man (OMIM) database. J Invest Dermatol 129 (2009), 2628–2636.
    • (2009) J Invest Dermatol , vol.129 , pp. 2628-2636
    • Feramisco, J.D.1    Sadreyev, R.I.2    Murray, M.L.3
  • 10
    • 43449084027 scopus 로고    scopus 로고
    • The classification of inherited epidermolysis bullosa (EB): report of the Third International Consensus Meeting on Diagnosis and Classification of EB
    • Fine, J.D., Eady, R.A., Bauer, E.A., et al. 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 (2008), 931–950.
    • (2008) J Am Acad Dermatol , vol.58 , pp. 931-950
    • Fine, J.D.1    Eady, R.A.2    Bauer, E.A.3
  • 11
    • 77956293326 scopus 로고    scopus 로고
    • Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice
    • Fujita, Y., Abe, R., Inokuma, D., et al. Bone marrow transplantation restores epidermal basement membrane protein expression and rescues epidermolysis bullosa model mice. Proc Natl Acad Sci USA 107 (2010), 14345–14350.
    • (2010) Proc Natl Acad Sci USA , vol.107 , pp. 14345-14350
    • Fujita, Y.1    Abe, R.2    Inokuma, D.3
  • 12
    • 69949112900 scopus 로고    scopus 로고
    • Adhesive stripping to remove epidermis in junctional epidermolysis bullosa for revertant cell therapy
    • Gostynski, A., Deviaene, F.C., Pasmooij, A.M., et al. Adhesive stripping to remove epidermis in junctional epidermolysis bullosa for revertant cell therapy. Br J Dermatol 161 (2009), 444–447.
    • (2009) Br J Dermatol , vol.161 , pp. 444-447
    • Gostynski, A.1    Deviaene, F.C.2    Pasmooij, A.M.3
  • 13
    • 0142084745 scopus 로고    scopus 로고
    • LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
    • Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302 (2003), 415–419.
    • (2003) Science , vol.302 , pp. 415-419
    • Hacein-Bey-Abina, S.1    Von Kalle, C.2    Schmidt, M.3
  • 14
    • 79955574166 scopus 로고    scopus 로고
    • Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy
    • Howden, S.E., Gore, A., Li, Z., et al. Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy. Proc Natl Acad Sci USA 108 (2011), 6537–6542.
    • (2011) Proc Natl Acad Sci USA , vol.108 , pp. 6537-6542
    • Howden, S.E.1    Gore, A.2    Li, Z.3
  • 15
    • 83155188960 scopus 로고    scopus 로고
    • Inherited epidermolysis bullosa: new diagnostic criteria and classification
    • Intong, L.R., Murrell, D.F., Inherited epidermolysis bullosa: new diagnostic criteria and classification. Clin Dermatol 30 (2012), 70–77.
    • (2012) Clin Dermatol , vol.30 , pp. 70-77
    • Intong, L.R.1    Murrell, D.F.2
  • 16
    • 79957696056 scopus 로고    scopus 로고
    • Generation of keratinocytes from normal and recessive dystrophic epidermolysis bullosa-induced pluripotent stem cells
    • Itoh, M., Kiuru, M., Cairo, M.S., et al. Generation of keratinocytes from normal and recessive dystrophic epidermolysis bullosa-induced pluripotent stem cells. Proc Natl Acad Sci USA 108 (2011), 8797–8802.
    • (2011) Proc Natl Acad Sci USA , vol.108 , pp. 8797-8802
    • Itoh, M.1    Kiuru, M.2    Cairo, M.S.3
  • 17
    • 0030975365 scopus 로고    scopus 로고
    • Revertant mosaicism in epidermolysis bullosa caused by mitotic gene conversion
    • Jonkman, M.F., Scheffer, H., Stulp, R., et al. Revertant mosaicism in epidermolysis bullosa caused by mitotic gene conversion. Cell 88 (1997), 543–551.
    • (1997) Cell , vol.88 , pp. 543-551
    • Jonkman, M.F.1    Scheffer, H.2    Stulp, R.3
  • 18
    • 70349755570 scopus 로고    scopus 로고
    • Achieving successful delivery of nucleic acids to skin: 6th Annual Meeting of the International Pachyonychia Congenita Consortium
    • Kaspar, R.L., McLean, W.H., Schwartz, M.E., Achieving successful delivery of nucleic acids to skin: 6th Annual Meeting of the International Pachyonychia Congenita Consortium. J Invest Dermatol 129 (2009), 2085–2087.
    • (2009) J Invest Dermatol , vol.129 , pp. 2085-2087
    • Kaspar, R.L.1    McLean, W.H.2    Schwartz, M.E.3
  • 19
    • 69949184884 scopus 로고    scopus 로고
    • Mechanisms of fibroblast cell therapy for dystrophic epidermolysis bullosa: high stability of collagen VII favors long-term skin integrity
    • Kern, J.S., Loeckermann, S., Fritsch, A., et al. Mechanisms of fibroblast cell therapy for dystrophic epidermolysis bullosa: high stability of collagen VII favors long-term skin integrity. Mol Ther 17 (2009), 1605–1615.
    • (2009) Mol Ther , vol.17 , pp. 1605-1615
    • Kern, J.S.1    Loeckermann, S.2    Fritsch, A.3
  • 20
    • 77952483707 scopus 로고    scopus 로고
    • Bone marrow stem cell therapy for recessive dystrophic epidermolysis bullosa
    • Kiuru, M., Itoh, M., Cairo, M.S., et al. Bone marrow stem cell therapy for recessive dystrophic epidermolysis bullosa. Dermatol Clin 28 (2010), 371–382.
    • (2010) Dermatol Clin , vol.28 , pp. 371-382
    • Kiuru, M.1    Itoh, M.2    Cairo, M.S.3
  • 21
    • 79952441928 scopus 로고    scopus 로고
    • Revertant mosaicism in skin: natural gene therapy
    • Lai-Cheong, J.E., McGrath, J.A., Uitto, J., Revertant mosaicism in skin: natural gene therapy. Trends Mol Med 7 (2011), 140–148.
    • (2011) Trends Mol Med , vol.7 , pp. 140-148
    • Lai-Cheong, J.E.1    McGrath, J.A.2    Uitto, J.3
  • 22
    • 76349084709 scopus 로고    scopus 로고
    • First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder
    • Leachman, S.A., Hickerson, R.P., Schwartz, M.E., et al. First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder. Mol Ther 18 (2010), 442–446.
    • (2010) Mol Ther , vol.18 , pp. 442-446
    • Leachman, S.A.1    Hickerson, R.P.2    Schwartz, M.E.3
  • 23
    • 79951513428 scopus 로고    scopus 로고
    • Zebrafish: a model system to study heritable skin diseases
    • Li, Q., Frank, M., Thisse, C.I., et al. Zebrafish: a model system to study heritable skin diseases. J Invest Dermatol 131 (2011), 565–571.
    • (2011) J Invest Dermatol , vol.131 , pp. 565-571
    • Li, Q.1    Frank, M.2    Thisse, C.I.3
  • 24
    • 33845524625 scopus 로고    scopus 로고
    • Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells
    • Mavilio, F., Pellegrini, G., Ferrari, S., et al. Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nat Med 12 (2006), 1397–1402.
    • (2006) Nat Med , vol.12 , pp. 1397-1402
    • Mavilio, F.1    Pellegrini, G.2    Ferrari, S.3
  • 25
    • 79954570064 scopus 로고    scopus 로고
    • The phenotypic and molecular genetic features of pachyonychia congenita
    • McLean, W.H., Hansen, C.D., Eliason, M.J., et al. The phenotypic and molecular genetic features of pachyonychia congenita. J Invest Dermatol 131 (2011), 1015–1017.
    • (2011) J Invest Dermatol , vol.131 , pp. 1015-1017
    • McLean, W.H.1    Hansen, C.D.2    Eliason, M.J.3
  • 26
    • 83155181586 scopus 로고    scopus 로고
    • Keratin disorders: from gene to therapy
    • McLean, W.H., Moore, C.B., Keratin disorders: from gene to therapy. Hum Mol Genet 20 (2011), R189–R197.
    • (2011) Hum Mol Genet , vol.20 , pp. R189-R197
    • McLean, W.H.1    Moore, C.B.2
  • 27
    • 79960316706 scopus 로고    scopus 로고
    • HBEGF induces COL7A1 expression in keratinocytes and fibroblasts: possible mechanism underlying allogeneic fibroblast therapy in recessive dystrophic epidermolysis bullosa
    • Nagy, N., Almaani, N., Tanaka, A., et al. HBEGF induces COL7A1 expression in keratinocytes and fibroblasts: possible mechanism underlying allogeneic fibroblast therapy in recessive dystrophic epidermolysis bullosa. J Invest Dermatol 131 (2011), 1771–1774.
    • (2011) J Invest Dermatol , vol.131 , pp. 1771-1774
    • Nagy, N.1    Almaani, N.2    Tanaka, A.3
  • 28
    • 70450245391 scopus 로고    scopus 로고
    • Animal models of epidermolysis bullosa
    • Natsuga, K., Shinkuma, S., Nishie, W., et al. Animal models of epidermolysis bullosa. Dermatol Clin 28 (2010), 137–142.
    • (2010) Dermatol Clin , vol.28 , pp. 137-142
    • Natsuga, K.1    Shinkuma, S.2    Nishie, W.3
  • 29
    • 84857708541 scopus 로고    scopus 로고
    • First symposium on natural gene therapy of the skin
    • Pasmooij, A.M., Jonkman, M.F., First symposium on natural gene therapy of the skin. Exp Dermatol 21 (2012), 236–239.
    • (2012) Exp Dermatol , vol.21 , pp. 236-239
    • Pasmooij, A.M.1    Jonkman, M.F.2
  • 30
    • 78649656327 scopus 로고    scopus 로고
    • Stem cell therapies for recessive dystrophic epidermolysis bullosa
    • Petrova, A., Ilic, D., McGrath, J.A., Stem cell therapies for recessive dystrophic epidermolysis bullosa. Br J Dermatol 163 (2010), 1149–1156.
    • (2010) Br J Dermatol , vol.163 , pp. 1149-1156
    • Petrova, A.1    Ilic, D.2    McGrath, J.A.3
  • 31
    • 58149250413 scopus 로고    scopus 로고
    • 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. Injection of recombinant human type VII collagen corrects the disease phenotype in a murine model of dystrophic epidermolysis bullosa. Mol Ther 17 (2009), 26–33.
    • (2009) Mol Ther , vol.17 , pp. 26-33
    • Remington, J.1    Wang, X.2    Hou, Y.3
  • 32
    • 77958109358 scopus 로고    scopus 로고
    • Long-term type VII collagen restoration to human epidermolysis bullosa skin tissue
    • Siprashvili, Z., Nguyen, N.T., Bezchinsky, M.Y., et al. Long-term type VII collagen restoration to human epidermolysis bullosa skin tissue. Hum Gene Ther 21 (2010), 1299–1310.
    • (2010) Hum Gene Ther , vol.21 , pp. 1299-1310
    • Siprashvili, Z.1    Nguyen, N.T.2    Bezchinsky, M.Y.3
  • 33
    • 37049039660 scopus 로고    scopus 로고
    • Development of therapeutic siRNAs for pachyonychia congenita
    • Smith, F.J.D., Hickerson, R.P., Sayers, J.M., et al. Development of therapeutic siRNAs for pachyonychia congenita. J Invest Dermatol 128 (2008), 50–58.
    • (2008) J Invest Dermatol , vol.128 , pp. 50-58
    • Smith, F.J.D.1    Hickerson, R.P.2    Sayers, J.M.3
  • 34
    • 0022381061 scopus 로고
    • Epidermolysis bullosa acquisita antigen is synthesized by both human keratinocytes and human dermal fibroblasts
    • Stanley, J.R., Rubinstein, N., Klaus-Kovtun, V., Epidermolysis bullosa acquisita antigen is synthesized by both human keratinocytes and human dermal fibroblasts. J Invest Dermatol 85 (1985), 542–545.
    • (1985) J Invest Dermatol , vol.85 , pp. 542-545
    • Stanley, J.R.1    Rubinstein, N.2    Klaus-Kovtun, V.3
  • 35
    • 67649836055 scopus 로고    scopus 로고
    • Molecular therapies for heritable blistering diseases
    • Tamai, K., Kaneda, Y., Uitto, J., Molecular therapies for heritable blistering diseases. Trends Mol Med 15 (2009), 285–292.
    • (2009) Trends Mol Med , vol.15 , pp. 285-292
    • Tamai, K.1    Kaneda, Y.2    Uitto, J.3
  • 36
    • 77955171264 scopus 로고    scopus 로고
    • SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa
    • Titeux, M., Pendaries, V., Zanta-Boussif, M.A., et al. SIN retroviral vectors expressing COL7A1 under human promoters for ex vivo gene therapy of recessive dystrophic epidermolysis bullosa. Mol Ther 18 (2010), 1509–1518.
    • (2010) Mol Ther , vol.18 , pp. 1509-1518
    • Titeux, M.1    Pendaries, V.2    Zanta-Boussif, M.A.3
  • 37
    • 79957595404 scopus 로고    scopus 로고
    • Concise review: transplantation of human hematopoietic cells for extracellular matrix protein deficiency in epidermolysis bullosa
    • Tolar, J., Blazar, B.R., Wagner, J.E., Concise review: transplantation of human hematopoietic cells for extracellular matrix protein deficiency in epidermolysis bullosa. Stem Cells 29 (2011), 900–906.
    • (2011) Stem Cells , vol.29 , pp. 900-906
    • Tolar, J.1    Blazar, B.R.2    Wagner, J.E.3
  • 38
    • 60849091919 scopus 로고    scopus 로고
    • Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells
    • Tolar, J., Ishida-Yamamoto, A., Riddle, M., et al. Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells. Blood 113 (2009), 1167–1174.
    • (2009) Blood , vol.113 , pp. 1167-1174
    • Tolar, J.1    Ishida-Yamamoto, A.2    Riddle, M.3
  • 39
    • 79952765436 scopus 로고    scopus 로고
    • Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa
    • Tolar, J., Xia, L., Riddle, M.J., et al. Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa. J Invest Dermatol 131 (2011), 848–856.
    • (2011) J Invest Dermatol , vol.131 , pp. 848-856
    • Tolar, J.1    Xia, L.2    Riddle, M.J.3
  • 40
    • 66749179401 scopus 로고    scopus 로고
    • Progress in heritable skin diseases: translational implications of mutation analysis and prospects of molecular therapies
    • Uitto, J., Progress in heritable skin diseases: translational implications of mutation analysis and prospects of molecular therapies. Acta Derm Venereol 89 (2009), 228–235.
    • (2009) Acta Derm Venereol , vol.89 , pp. 228-235
    • Uitto, J.1
  • 41
    • 79960304558 scopus 로고    scopus 로고
    • Cell-based therapy for RDEB: how does it work?
    • Uitto, J., Cell-based therapy for RDEB: how does it work?. J Invest Dermatol 131 (2011), 1597–1599.
    • (2011) J Invest Dermatol , vol.131 , pp. 1597-1599
    • Uitto, J.1
  • 42
    • 79952758979 scopus 로고    scopus 로고
    • Regenerative medicine for skin diseases: iPS cells to the rescue
    • Uitto, J., Regenerative medicine for skin diseases: iPS cells to the rescue. J Invest Dermatol 131 (2011), 812–814.
    • (2011) J Invest Dermatol , vol.131 , pp. 812-814
    • Uitto, J.1
  • 43
    • 84856915092 scopus 로고    scopus 로고
    • Novel molecular therapies for heritable skin disorders
    • Uitto, J., Christiano, A.M., McLean, W.H., et al. Novel molecular therapies for heritable skin disorders. J Invest Dermatol 132 (2012), 820–828.
    • (2012) J Invest Dermatol , vol.132 , pp. 820-828
    • Uitto, J.1    Christiano, A.M.2    McLean, W.H.3
  • 44
    • 77956621310 scopus 로고    scopus 로고
    • Bone marrow transplantation for recessive dystrophic epidermolysis bullosa
    • Wagner, J.E., Ishida-Yamamoto, A., McGrath, J.A., et al. Bone marrow transplantation for recessive dystrophic epidermolysis bullosa. N Engl J Med 363 (2010), 629–639.
    • (2010) N Engl J Med , vol.363 , pp. 629-639
    • Wagner, J.E.1    Ishida-Yamamoto, A.2    McGrath, J.A.3
  • 45
    • 49549096936 scopus 로고    scopus 로고
    • Potential of fibroblast cell therapy for recessive dystrophic epidermolysis bullosa
    • Wong, T., Gammon, L., Liu, L., et al. Potential of fibroblast cell therapy for recessive dystrophic epidermolysis bullosa. J Invest Dermatol 128 (2008), 2179–2189.
    • (2008) J Invest Dermatol , vol.128 , pp. 2179-2189
    • Wong, T.1    Gammon, L.2    Liu, L.3
  • 46
    • 0242658535 scopus 로고    scopus 로고
    • Normal and gene-corrected dystrophic epidermolysis bullosa fibroblasts alone can produce type VII collagen at the basement membrane zone
    • Woodley, D.T., Krueger, G.G., Jorgensen, C.M., et al. Normal and gene-corrected dystrophic epidermolysis bullosa fibroblasts alone can produce type VII collagen at the basement membrane zone. J Invest Dermatol 121 (2003), 1021–1028.
    • (2003) J Invest Dermatol , vol.121 , pp. 1021-1028
    • Woodley, D.T.1    Krueger, G.G.2    Jorgensen, C.M.3
  • 47
    • 77952505237 scopus 로고    scopus 로고
    • Fibroblast-based cell therapy strategy for recessive dystrophic epidermolysis bullosa
    • Yan, W.F., Murrell, D.F., Fibroblast-based cell therapy strategy for recessive dystrophic epidermolysis bullosa. Dermatol Clin 28 (2010), 367–370.
    • (2010) Dermatol Clin , vol.28 , pp. 367-370
    • Yan, W.F.1    Murrell, D.F.2
  • 48
    • 84879690707 scopus 로고    scopus 로고
    • Molecular therapeutics for heritable skin diseases
    • Uitto, J., Molecular therapeutics for heritable skin diseases. J Invest Dermatol 132 (2012), E29–E34.
    • (2012) J Invest Dermatol , vol.132 , pp. E29-E34
    • Uitto, J.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.