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Postulated mechanism by which fibroblast therapy may ameliorate the blistering tendency in recessive dystrophic epidermolysis bullosa (RDEB). (a) In normal skin, keratinocytes synthesize type VII collagen molecules (red), which assemble into anchoring fibrils. These fibrils entrap the interstitial collagen fibers in the dermis, securing the stable association at the dermal–epidermal junction. (b) In some patients with RDEB, there are only a few rudimentary anchoring fibrils, allowing formation of blisters below the lamina densa as a result of minor trauma. (c) Allogeneic fibroblasts injected directly into the dermis elicit a subclinical immune reaction that leads to synthesis of heparin-binding EGF-like growth factor (HB-EGF), which upregulates the synthesis and assembly of the patient's own mutated type VII collagen. The increase in the rudimentary anchoring fibrils, which are partially functional, stabilizes the association of epidermis to the underlying dermis and ameliorates the blistering tendency (adapted from Uitto, 2011a)
 

Principios de las dos estrategias para la transferencia de genes (a) estrategia in  vivo, los genes pueden ser transferidos directamente a la piel mediante aplicación tópica, inyección intracut´neo o por bombardeo de partículas biolisticas (pistola de genes) (b) estrategia ex vivo. Las células diana como los queratinocitos se extraen de la piel y se cultivan. Las células son entonces transducidas con el vector expresando el transgen y ls células transgenicas son seleccionadas y crecen en laminas epiteliales que entonces pueden ser injertadas en el donante original

Trends in molecular medicine 2009 15:285.