161
Views
3
CrossRef citations to date
0
Altmetric
Editorial

Cutaneous gene therapy for haemophilia

Pages 763-766 | Published online: 23 Feb 2005

References

  • KAZAZIAN HH JR TUDDENHAM EGD, ANTONARAKIS SE: Hemophilia A: deficiency of coagulation Factor VIII. In: The Metabolic and Molecular Bases of Inherited Disease. 8th edition. Scriver C, Beaudet A, Sly W, Valle D (Eds), McGraw-Hill, New York, USA (2001):4367–4392.
  • •A thorough review of the molecular, genetic and clinical features of haemophilia A.
  • POLLACK ES, HIGH KA: Hemophilia B: Factor IX deficiency. In: The Metabolic and Molecular Bares of Inherited Disease. 8th edition. Scriver C, Beaudet A, Sly W, Valle D (Eds), McGraw-Hill, New York, USA (2001):4393–4413.
  • •A thorough review of the molecular, genetic and clinical features of haemophilia B.
  • GREENHALGH D, ROTHNAGEL J, ROOP D: Epidermis: an attractive target tissue for gene therapy. Invert. Dermatol (1994) 103:63S–69S.
  • VASSAR R, ROSENBERG M, ROSS S, TYNER A, FUCHS E: Tissue-specific and differentiation-specific expression of a human K14 keratin gene in transgenic mice. Proc. Nati Acad. Sci. USA (1989) 86:1563–1567.
  • CARROLL J, ALBERS K, GARLICK J, HARRINGTON R, TAICHMAN L: Tissue- and stratum-specific expression of the human involucrin promoter in transgenic mice. Proc. Nati Acad. Sci. USA (1993) 90:10270–110274.
  • BOYCE S: Epidermis as a secretory tissue. Invert. Dermatol (1994) 102:8–10.
  • GERRARD AJ, HUDSON DL, BROWNLEE GG, WATT FM: Towards gene therapy for haemophilia B using primary human keratinocytes. Nat. Genet. (1993) 3:180–183.
  • ••The first study demonstrating thatFactor IX can enter the circulation from transplanted keratinocytes modified to express Factor IX.
  • ALEXANDER MY, BIDICHANDANI SI, COUSINS FM, ROBINSON CJ, DUFFIE E, AKHURST RJ: Circulating human Factor IX in keratin-promoter transgenic mice: a feasibility study for gene therapy of hemophilia B. Hum. Ma Genet. (1995) 4:993–999.
  • PAGE SM, BROWNLEE GG: An ex vivokeratinocyte model for gene therapy of hemophilia B. Invest. Dermatol (1997)109:139–145.
  • WHITE SJ, PAGE SM, MARGARITAS P, BROWNLEE G: Long-term expression of human clotting Factor IX from retrovirally transduced primary human keratinocytes in vivo. Hum. Gene Their (1998) 9:1187–1195.
  • •A study demonstrating sustained Factor IX expression in mice engrafted with Factor IX-expressing keratinocytes.
  • FAKHARZADEH SS, ZHANG Y, SARKAR R, KAZAZIAN HH JR: Correction of the coagulation defect in hemophilia A mice through Factor VIII expression in skin. Blood (2000) 95:2799–2805.
  • ••The first study to demonstrate thatepidermal expression of a coagulation factor can achieve phenotypic correction in an animal model for haemophilia.
  • CHEUNG WE VAN DEN BORN J, KUHN K, KJELLEN L, HUDSON BG, STAFFORD DW: Identification of the endothelial cell binding site for Factor IX. Proc. Natl Acad. Sci. USA (1996) 93:11068–11073.
  • HERZOG RW, HAGSTROM JN, KUNG SH et al.: Stable gene transfer and expression of human blood coagulation Factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl. Acad. Sci. USA (1997) 94:5804–5809.
  • GUI T, LIN HF, JIN DY et al.: Circulating and binding characteristics of wild-type Factor IX and certain Gla domain mutants in vivo. Blood (2002) 100:153–158.
  • SCALLAN CD, LIU T, PARKER AE et al: Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII. Blood (2003) 102:3919–3926.
  • PIPE S, KAUFMAN R: Characterization of a genetically engineered inactivation-resistant coagulation Factor VIII. Proc. Nati Acad. Sci. USA (1997) 94:11851–11856.
  • MIAO HZ, SIRACHAINAN N, PALMER L et al.: Bioengineering of coagulation Factor VIII for improved secretion. Blood (2004) 103 (9) :3412–3419.
  • LIN MT, PULKKINEN L, UITTO J, YOON K: The gene gun: current applications in cutaneous gene therapy. hat. Dermatol (2000) 39:161–170.
  • TREZISE AE, PALAZON L, DAVIES WL, COLLEDGE WH: In vivo gene expression: DNA electrotransfer. Carr: Opin. Mol. Ther. (2003) 5:397–404.
  • HENGGE UR, CHAN EF, FOSTER RA, WALKER PS, VOGEL JC: Cytokine gene expression in epidermis with biological effects following injection of naked DNA. Nat. Genet. (1995) 10:161–166.
  • GHAZIZADEH S, HARRINGTON R, TAICHMAN L: In vivo transduction of mouse epidermis with recombinant retroviral vectors: implications for cutaneous gene therapy. Gene The]: (1999) 6:1267-1275. AffiliationSteven S Fakharzadeh MD, PhD Department of Dermatology, University of Pennsylvania Health System, 415 Curie Boulevard, Room 235 B, Clinical Research Building, Philadelphia, Pennsylvania 19104–6142, USA Tel: +1 215 573 5709; Fax: +1 215 573 2143; E-mail: [email protected]

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.