Valutazione dell'efficienza, efficacia e sicurezza di vettori lentivirali nel trasferimento del gene CFTR in sistemi modello di epitelio respiratorio in fibrosi cistica

One of the possible strategies for therapy of Cystic Fibrosis is based on gene therapy. Gene therapy goal is to provide a normal copy of CFTR gene to defective tissues by using different gene transfer agents. HIV-1 derived vectors allow a prolonged expression of therapeutic gene and they are able to infect quiescent cells like respiratory epithelium cells. Primary goal of the project is concerned with the realization of a lentivirus working live agent, transferring CFTR WT gene, or SHRNA molecules directed against ENAC subunits; ENAC is a sodium channel hyperactive in cystic fibrosis.