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  • Casal ML, Lewis JR, Mauldin EA, Tardivel A, Ingold K, Favre M, Paradies F, Demotz S, Gaide O, Schneider P. 2007. Significant correction of disease after postnatal administration of recombinant ectodysplasin A in canine X-linked ectodermal dysplasia. Am J Hum Genet 81: 10501056.
  • Cavazzana-Calvo M, Fischer A. 2004. Efficacy of gene therapy for SCID is being confirmed. Lancet 364: 21552156.
  • Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, Bousso P, Deist FL, Fischer A. 2000. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288: 669672.
  • Davies JC, Alton EW, Bush A. 2007. Cystic fibrosis. Br Med J 335: 12551259.
  • Deichmann A, Hacein-Bey-Abina S, Schmidt M, Garrigue A, Brugman MH, Hu J, Glimm H, Gyapay G, Prum B, Fraser CC, Fischer N, Schwarzwaelder K, Siegler ML, de Ridder D, Pike-Overzet K, Howe SJ, Thrasher AJ, Wagemaker G, Abel U, Staal FJ, Delabesse E, Villeval JL, Aronow B, Hue C, Prinz C, Wissler M, Klanke C, Weissenbach J, Alexander I, Fischer A, von Kalle C, Cavazzana-Calvo M. 2007. Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest 117: 22252232.
  • Fischer A, Le Deist F, Hacein-Bey-Abina S, André-Schmutz I, de Saint Basile G, de Villartay JP, Cavazzana-Calvo M. 2005. Severe combined immunodeficiency. A model disease for molecular immunology and therapy. Immunol Rev 203: 98109.
  • Gaide O, Schneider P. 2003. Permanent correction of an inherited ectodermal dysplasia with recombinant EDA. Nat Med 9: 614618.
  • GeMCRIS. 2009. http://www.gemcris.od.nih.gov/.
  • Irvine A. 2006. Ectodermal dysplasias. In: Harper J, Oranje A, Prose N, editors. Textbook of pediatric dermatology. London: Wiley-Blackwell. pp 14121466.
  • Itin PH, Fistarol SK. 2004. Ectodermal dysplasias. Am J Med Genet Part C 131C: 4551.
  • Koster MI, Roop DR. 2004. The role of p63 in development and differentiation of the epidermis. J Dermatol Sci 34: 39.
  • Lucky AW, Palisson F, Mellerio JE. 2008. The IVth International Symposium on Epidermolysis Bullosa, Santiago, Chile, 27–29 September 2007. J Dermatol Sci 49: 178184.
  • Mavilio F, Pellegrini G, Ferrari S, Di Nunzio F, Di Iorio E, Recchia A, Maruggi G, Ferrari G, Provasi E, Bonini C, Capurro S, Conti A, Magnoni C, Giannetti A, De Luca M. 2006. Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nat Med 12: 13971402.
  • Niederau C, vom Dahl S, Haussinger D. 1998. First long-term results of imiglucerase therapy of type 1 Gaucher disease. Eur J Med Res 3: 2530.
  • Rosenecker J, Huth S, Rudolph C. 2006. Gene therapy for cystic fibrosis lung disease: Current status and future perspectives. Curr Opin Mol Ther 8: 439445.
  • Russell CS, Clarke LA. 1999. Recombinant proteins for genetic disease. Clin Genet 55: 389394.
  • Ting JP, Kastner DL, Hoffman HM. 2006. CATERPILLERs, pyrin and hereditary immunological disorders. Nat Rev Immunol 6: 183195.
  • Zonana J. 2008. EDA (ED1), EDAR, EDARADD: Hypohidrotic ectodermal dysplasias and the ectodysplasin signaling pathway. In: Epstein C, Erickson R, Wynshaw-Boris A, editors. Inborn Errors of Development: The Molecular Basis of Clinical Disorders of Morphogenesis. San Francisco: Oxford University Press. p 442448.