González-Ramos IA, Jaloma-Cruz AR

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

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.

REFERENCES

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