title: [The skin as a vehicle for gene therapy: hemophilia B, an application model].
abstract: Artificial skin offers important advantages in gene therapy tor 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.
authors.affiliation: División de Genética, Centro de Investigación Biomédica de Occidente, Instituto Mexicano del Seguro Social, Mexico.|Centro Universitario de Ciencias de la Salud, Universidad de Guadalajara, Mexico.
authors.forename: Isaura Araceli
authors.initials: IA
authors.lastname: González-Ramos
authors.affiliation: División de Genética, Centro de Investigación Biomédica de Occidente, Instituto Mexicano del Seguro Social, Mexico.
authors.forename: Ana Rebeca
authors.initials: AR
authors.lastname: Jaloma-Cruz
mesh_terms: D015316:Genetic Therapy / Q000379:methods*; D002836:Hemophilia B / Q000235:genetics / Q000628:therapy*; D006801:Humans; D012871:Skin Diseases / Q000235:genetics / Q000628:therapy; D017768:Skin, Artificial