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Evaluation of the use of keratinocytes sheets and ex vivo gene therapy in the treatment of epidermolysis bollosa  

 

The aim of the study is the production of neo-skin formed by sheets of keratinocytes and / or fibroblasts treated by ex vivo gene therapy for the potential treatment of patients with epidermolysis bullosa.

The execution of this sperimentation would lead solid foundation for the treatment of a severely disabling disease from the perspective of both organic and social.

The study is divide into five main phases:

  1. phase of biopsy on patients with epidermolysis bullosa, for the selection of  autologous keratinocytes / fibroblasti
  2. phase of gene therapy through retroviral carrier
  3. phase of amplification through keratinocytes / fibroblasti culture in vitro
  4. phase of  histological analysis of the results
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