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>Journals >Gaceta Médica de México >Year 2015, Issue 2


González-Ramos IA, Jaloma-Cruz AR
The skin as a vehicle for gene therapy: hemophilia B, an application model
Gac Med Mex 2015; 151 (2)

Language: Español
References: 5
Page: 266-269
PDF: 196.52 Kb.


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ABSTRACT

Artificial skin offers important advantages in gene therapy for its biosafety and simple monitoring. An easy access of keratinocytes through small biopsies and their in vitro expansion enriched with epithelial stem cells, make them an ideal target for long-term therapeutic transgene expression. Corrective cutaneous gene therapy has been recently applied in clinical trials on dermatological genetic diseases. In systemic monogenic diseases such as hemophilia B, the graft of genetically modified skin in murine experimental models has achieved a modest increase of clotting factor IX in plasma that may attenuate severe symptoms of the disease.


Key words: Artificial skin, Dermoepidermal equivalent, Cutaneous gene therapy, Hemophilia B.


REFERENCIAS

  1. Del Río M, Gache Y, Jorcano JL, Meneguzzi G, Larcher F. Current approaches and perspectives in human keratinocyte-based gene therapies. Gene Ther. 2004;11 Suppl 1:S57-63.

  2. Llames SG, Del Rio M, Larcher F, et al. Human plasma as a dermal scaffold for the generation of a completely autologous bioengineered skin. Transplantation. 2004;77(3):350-5.

  3. Martínez-Santamaría L, Guerrero-Aspizua S, Del Río M. Bioingeniería cutánea: aplicaciones preclínicas y clínicas. Actas Dermosifiliogr. 2012;103:5-11.

  4. Gerrard AJ, Austen DEJG, Brownlee GG. Recombinant factor IX secreted by transduced human keratinocytes is biologically active. Br J Haematol. 1996;95(3):561-3.

  5. White SJ, Page SM, Margaritis P, Brownlee GG. Long-term expressions of human clotting factor IX from retrovirally transduced primary human keratinocytes in vivo. Hum Gene Ther. 1998;9(8):1187-95.






>Journals >Gaceta Médica de México >Year 2015, Issue 2
 

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