Cell-and protein-based therapy approaches for epidermolysis bullosa

Methods in Molecular Biology

Volumn 961, Issue , 2013, Pages 425-440

  Nyström, Alexander ^a   Bruckner Tuderman, Leena ^a,b   Kern, Johannes S ^a  

^a UNIVERSITY OF FREIBURG   (Germany)

^b UNIVERSITY OF FREIBURG   (Germany)

Author keywords

Collagen VII; Dystrophic epidermolysis bullosa; Fibroblast; Recombinant protein purification

Indexed keywords

COLLAGEN TYPE 7; RECOMBINANT PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL MIGRATION; CELL THERAPY; CONTROLLED STUDY; EPIDERMOLYSIS BULLOSA; FEASIBILITY STUDY; FIBROBLAST CULTURE; IMMUNOFLUORESCENCE; MOUSE; NONHUMAN; PRIORITY JOURNAL; SKIN FIBROBLAST; STAINING; ANIMAL; BIOLOGICAL THERAPY; CELL CULTURE; CULTURE TECHNIQUE; DISEASE MODEL; EPIDERMOLYSIS BULLOSA DYSTROPHICA; FIBROBLAST; FLUORESCENT ANTIBODY TECHNIQUE; HUMAN; INTRADERMAL DRUG ADMINISTRATION; ISOLATION AND PURIFICATION; METABOLISM; METHODOLOGY; PATHOLOGY; SKIN; TRANSPLANTATION;

ANIMALS; CELL CULTURE TECHNIQUES; CELLS, CULTURED; COLLAGEN TYPE VII; DISEASE MODELS, ANIMAL; EPIDERMOLYSIS BULLOSA DYSTROPHICA; FIBROBLASTS; FLUORESCENT ANTIBODY TECHNIQUE; HUMANS; INJECTIONS, INTRADERMAL; MICE; RECOMBINANT PROTEINS; SKIN; TISSUE THERAPY;

MLCS; MLOWN;

EID: 84879314673     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-62703-227-8_29     Document Type: Article

References (25)

1. Bruckner-Tuderman L (2009) Can type VII collagen injections cure dystrophic epidermolysis bullosa? Mol Ther 17:6-7
2. Bruckner-Tuderman L (2010) Systemic therapy for a genetic skin disease. N Engl J Med 363:680-682
3. Uitto J, McGrath JA, Rodeck U, Bruckner-Tuderman L, Robinson EC (2010) Progress in epidermolysis bullosa research: toward treatment and cure. J Invest Dermatol 130: 1778-1784
4. Bruckner-Tuderman L (2010) Dystrophic epidermolysis bullosa: pathogenesis and clinical features. Dermatol Clin 28:107-114
5. Fritsch A, Loeckermann S, Kern JS, Braun A, Bosl MR, Bley TA, Schumann H, von Elverfeldt D, Paul D, Erlacher M, Berens von Rautenfeld D, Hausser I, Fassler R, Bruckner-Tuderman L (2008) A hypomorphic mouse model of dystrophic epidermolysis bullosa reveals mechanisms of disease and response to fibroblast therapy. J Clin Invest 118:1669-1679
6. Ito K, Sawamura D, Goto M, Nakamura H, Nishie W, Sakai K, Natsuga K, Shinkuma S, Shibaki A, Uitto J, Denton CP, Nakajima O, Akiyama M, Shimizu H (2009) Keratinocyte-/fibroblast-targeted rescue of Col7a1-disrupted mice and generation of an exact dystrophic epidermolysis bullosa model using a human COL7A1 mutation. Am J Pathol 175:2508-2517
7. Woodley DT, Krueger GG, Jorgensen CM, Fairley JA, Atha T, Huang Y, Chan L, Keene DR, Chen M (2003) Normal and gene-corrected dystrophic epidermolysis bullosa fibroblasts alone can produce type VII collagen at the basement membrane zone. J Invest Dermatol 121:1021-1028
8. Haniffa MA, Wang XN, Holtick U, Rae M, Isaacs JD, Dickinson AM, Hilkens CM, Collin MP (2007) Adult human fibroblasts are potent immunoregulatory cells and functionally equivalent to mesenchymal stem cells. J Immunol 179:1595-1604
9. Kern JS, Loeckermann S, Fritsch A, Hausser I, Roth W, Magin TM, Mack C, Muller ML, Paul O, Ruther P, Bruckner-Tuderman L (2009) Mechanisms offibroblast cell therapy for dystrophic epidermolysis bullosa: high stability of collagen VII favors long-term skin integrity. Mol Ther 17:1605-1615
10. Nagy N, Almaani N, Tanaka A, Lai-Cheong JE, Techanukul T, Mellerio JE, McGrath JA (2011) HB-EGF induces COL7A1 expression in keratinocytes and fibroblasts: possible mechanism underlying allogeneic fibroblast therapy in recessive dystrophic epidermolysis bullosa. J Invest Dermatol 131:1771-1774
11. Wong T, Gammon L, Liu L, Mellerio JE, Dopping-Hepenstal PJ, Pacy J, Elia G, Jeffery R, Leigh IM, Navsaria H, McGrath JA (2008) Potential offibroblast cell therapy for recessive dystrophic epidermolysis bullosa. J Invest Dermatol 128:2179-2189
12. Woodley DT, Remington J, Huang Y, Hou Y, Li W, Keene DR, Chen M (2007) Intravenously injected human fibroblasts home to skin wounds, deliver type VII collagen, and promote wound healing. Mol Ther 15:628-635
13. Yan WF, Murrell DF (2010) Fibroblast-based cell therapy strategy for recessive dystrophic epidermolysis bullosa. Dermatol Clin 28:367-370, xii
14. Petrache I, Hajjar J, Campos M (2009) Safety and efficacy of alpha-1-antitrypsin augmentation therapy in the treatment of patients with alpha-1-antitrypsin deficiency. Biologics 3: 193-204
15. Parikh N, Riedl MA (2011) New therapeutics in C1iNH deficiency: a review of recent studies and advances. Curr Allergy Asthma Rep 11:300-308
16. Remington J, Wang X, Hou Y, Zhou H, Burnett J, Muirhead T, Uitto J, Keene DR, Woodley DT, Chen M (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
17. Woodley DT, Keene DR, Atha T, Huang Y, Lipman K, Li W, Chen M (2004) Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa. Nat Med 10:693-695
18. Fritsch A, Spassov S, Elfert S, Schlosser A, Gache Y, Meneguzzi G, Bruckner-Tuderman L (2009) Dominant-negative effects of COL7A1 mutations can be rescued by controlled overexpression of normal collagen VII. J Biol Chem 284:30248-30256
19. Heinonen S, Mannikko M, Klement JF, Whitaker-Menezes D, Murphy GF, Uitto J (1999) Targeted inactivation of the type VII collagen gene (Col7a1) in mice results in severe blistering phenotype: a model for recessive dystrophic epidermolysis bullosa. J Cell Sci 112(Pt 21):3641-3648
20. Hammami-Hauasli N, Raghunath M, Kuster W, Bruckner-Tuderman L (1998) Transient bullous dermolysis of the newborn associated with compound heterozygosity for recessive and dominant COL7A1 mutations. J Invest Dermatol 111:1214-1219
21. Mecklenbeck S, Compton SH, Mejia JE, Cervini R, Hovnanian A, Bruckner-Tuderman L, Barrandon Y (2002) A microinjected COL7A1-PAC vector restores synthesis of intact procollagen VII in a dystrophic epidermolysis bullosa keratinocyte cell line. Hum Gene Ther 13:1655-1662
22. Bruckner P, Prockop DJ (1981) Proteolytic enzymes as probes for the triple-helical conformation of procollagen. Anal Biochem 110:360-368
23. Goto M, Sawamura D, Ito K, Abe M, Nishie W, Sakai K, Shibaki A, Akiyama M, Shimizu H (2006) Fibroblasts show more potential as target cells than keratinocytes in COL7A1 gene therapy of dystrophic epidermolysis bullosa. J Invest Dermatol 126:766-772
24. Bruckner-Tuderman L, Schnyder UW, Winterhalter KH, Bruckner P (1987) Tissue form of type VII collagen from human skin and dermal fibroblasts in culture. Eur J Biochem 165:607-611
25. Chen M, Costa FK, Lindvay CR, Han YP, Woodley DT (2002) The recombinant expression offull-length type VII collagen and characterization of molecular mechanisms underlying dystrophic epidermolysis bullosa. J Biol Chem 277:2118-2124