Protein therapeutics for junctional epidermolysis bullosa: Incorporation of recombinant β3 chain into laminin 332 in β3-/- keratinocytes in vitro

Journal of Investigative Dermatology

Volumn 128, Issue 6, 2008, Pages 1476-1486

  Igoucheva, Olga ^a   Kelly, Aislinn ^a   Uitto, Jouni ^a   Alexeev, Vitali ^a  

^a THOMAS JEFFERSON UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

KALININ; POLYPEPTIDE; RECOMBINANT BETA 3 POLYPEPTIDE;

ARTICLE; BASEMENT MEMBRANE; CELL BASED GENE THERAPY; CELL CULTURE; CONFOCAL MICROSCOPY; DRUG PURIFICATION; DRUG SYNTHESIS; ENDOPLASMIC RETICULUM; EPIDERMOLYSIS BULLOSA; GENE TARGETING; HUMAN; HUMAN CELL; HUMAN TISSUE; IMMUNOFLUORESCENCE MICROSCOPY; KERATINOCYTE; PRIORITY JOURNAL; PROTEIN ASSEMBLY; PROTEIN SECRETION; WESTERN BLOTTING;

EID: 43749107571     PISSN: 0022202X     EISSN: 15231747     Source Type: Journal    
DOI: 10.1038/sj.jid.5701197     Document Type: Article

References (31)

1. M.J. Arin D.R. Roop Disease model: heritable skin blistering Trends Mol Med 7 2001 422 424
2. M. Aumailley L. Bruckner-Tuderman W.G. Carter R. Deutzmann D. Edgar P. Ekblom A simplified laminin nomenclature Matrix Biol 24 2005 326 332
3. S.E. Baker S.B. Hopkinson M. Fitchmun G.L. Andreason F. Frasier G. Plopper Laminin-5 and hemidesmosomes: role of the alpha 3 chain subunit in hemidesmosome stability and assembly J Cell Sci 109 Part 10 1996 2509 2520
4. N. Bendifallah F.W. Rasmussen V. Zachar P. Ebbesen P.E. Nielsen U. Koppelhus Evaluation of cell-penetrating peptides (CPPs) as vehicles for intracellular delivery of antisense peptide nucleic acid (PNA) Bioconjug Chem 17 2006 750 758
5. R. Brittingham J. Uitto A. Fertala High-affinity binding of the NC1 domain of collagen VII to laminin 5 and collagen IV Biochem Biophys Res Commun 343 2006 692 699
6. W.G. Carter P. Kaur S.G. Gil P.J. Gahr E.A. Wayner Distinct functions for integrins alpha 3 beta 1 in focal adhesions and alpha 6 beta 4/bullous pemphigoid antigen in a new stable anchoring contact (SAC) of keratinocytes: relation to hemidesmosomes J Cell Biol 111 1990 3141 3154
7. M. Chen M.P. Marinkovich A. Veis X. Cai C.N. Rao E.A. O'Toole Interactions of the amino-terminal noncollagenous (NC1) domain of type VII collagen with extracellular matrix components. A potential role in epidermal–dermal adherence in human skin J Biol Chem 272 1997 14516 14522
8. J.J. de Poorter K.S. Lipinski R.G. Nelissen T.W. Huizinga R.C. Hoeben Optimization of short-term transgene expression by sodium butyrate and ubiquitous chromatin opening elements (UCOEs) J Gene Med 9 2007 639 648
9. E. Dellambra J. Vailly G. Pellegrini S. Bondanza O. Golisano C. Macchia Corrective transduction of human epidermal stem cells in laminin-5-dependent junctional epidermolysis bullosa Hum Gene Ther 9 1998 1359 1370
10. J. Engel Laminins and other strange proteins Biochemistry 31 1992 10643 10651
11. C. Featherstone J. Uitto Ex vivo gene therapy cures a blistering skin disease Trends Mol Med 13 2007 219 222
12. J.D. Fine R.A. Eady E.A. Bauer R.A. Briggaman L. Bruckner-Tuderman A. Christiano Revised classification system for inherited epidermolysis bullosa: report of the Second International Consensus Meeting on diagnosis and classification of epidermolysis bullosa J Am Acad Dermatol 42 2000 1051 1066
13. J.B. Kim J.S. Choi K. Nam M. Lee J.S. Park J.K. Lee Enhanced transfection of primary cortical cultures using arginine-grafted PAMAM dendrimer, PAMAM-Arg J Control Release 114 2006 110 117
14. C. Krejsa M. Rogge W. Sadee Protein therapeutics: new applications for pharmacogenetics Nat Rev Drug Discov 5 2006 507 521
15. J.E. Kuster M.H. Guarnieri J.G. Ault L. Flaherty P.J. Swiatek IAP insertion in the murine LamB3 gene results in junctional epidermolysis bullosa Mamm Genome 8 1997 673 681
16. C. Matsui C.F. Nelson G.T. Hernandez G.S. Herron E.A. Bauer W.K. Hoeffler Gamma 2 chain of laminin-5 is recognized by monoclonal antibody GB3 J Invest Dermatol 105 1995 648 652
17. C. Matsui P. Pereira C.K. Wang C.F. Nelson T. Kutzkey C. Lanigan Extent of laminin-5 assembly and secretion effect junctional epidermolysis bullosa phenotype J Exp Med 187 1998 1273 1283
18. C. Matsui C.K. Wang C.F. Nelson E.A. Bauer W.K. Hoeffler The assembly of laminin-5 subunits J Biol Chem 270 1995 23496 23503
19. F. Mavilio G. Pellegrini S. Ferrari F. Di Nunzio E. Di Iorio A. Recchia Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells Nat Med 12 2006 1397 1402
20. D.R. McClay G.M. Wessel R.B. Marchase Intercellular recognition: quantitation of initial binding events Proc Natl Acad Sci USA 78 1981 4975 4979
21. X. Meng J.F. Klement D.A. Leperi D.E. Birk T. Sasaki R. Timpl Targeted inactivation of murine laminin gamma2-chain gene recapitulates human junctional epidermolysis bullosa J Invest Dermatol 121 2003 720 731
22. W.W. Nichols D.G. Murphy V.J. Cristofalo L.H. Toji A.E. Greene S.A. Dwight Characterization of a new human diploid cell strain, IMR-90 Science 196 1977 60 63
23. B.J. Nickoloff R.S. Mitra B.L. Riser V.M. Dixit J. Varani Modulation of keratinocyte motility. Correlation with production of extracellular matrix molecules in response to growth promoting and antiproliferative factors Am J Pathol 132 1988 543 551
24. T. Nishiyama S. Amano M. Tsunenaga K. Kadoya A. Takeda E. Adachi The importance of laminin 5 in the dermal–epidermal basement membrane J Dermatol Sci 24 Suppl 1 2000 S51 S59
25. P.B. Robbins Q. Lin J.B. Goodnough H. Tian X. Chen P.A. Khavari In vivo restoration of laminin 5 beta 3 expression and function in junctional epidermolysis bullosa Proc Natl Acad Sci USA 98 2001 5193 5198
26. P.B. Robbins S.M. Sheu J.B. Goodnough P.A. Khavari Impact of laminin 5 beta3 gene versus protein replacement on gene expression patterns in junctional epidermolysis bullosa Hum Gene Ther 12 2001 1443 1448
27. J. Vailly L. Gagnoux-Palacios E. Dell'Ambra C. Romero M. Pinola G. Zambruno Corrective gene transfer of keratinocytes from patients with junctional epidermolysis bullosa restores assembly of hemidesmosomes in reconstructed epithelia Gene Therapy 5 1998 1322 1332
28. R. Varki S. Sadowski E. Pfendner J. Uitto Epidermolysis bullosa. I. Molecular genetics of the junctional and hemidesmosomal variants J Med Genet 43 2006 641 652
29. R. Varki S. Sadowski J. Uitto E. Pfendner Epidermolysis bullosa. II. Type VII collagen mutations and phenotype–genotype correlations in the dystrophic subtypes J Med Genet 44 2007 181 192
30. S. Williams T. Mustoe T. Mulcahy M. Griffiths D. Simpson M. Antoniou CpG-island fragments from the HNRPA2B1/CBX3 genomic locus reduce silencing and enhance transgene expression from the hCMV promoter/enhancer in mammalian cells BMC Biotechnol 5 2005 17
31. D.T. Woodley D.R. Keene T. Atha Y. Huang K. Lipman W. Li Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa Nat Med 10 2004 693 695