2026-09-24T07:10:19Zhttps://iris.unica.it/oai/requestoai:iris.unica.it:11584/2659532025-06-13T02:22:38Zcom_11584_207615com_11584_111066col_11584_265854
Valutazione dell'efficienza, efficacia e sicurezza di vettori lentivirali nel trasferimento del gene CFTR in sistemi modello di epitelio respiratorio in fibrosi cistica
CASTELLANI, STEFANO
CFTR
ENAC.
Fibrosi cistica
Lentivirus
Terapia genica
Settore MED/38 - Pediatria Generale e Specialistica
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.
2007-12-14
info:eu-repo/semantics/doctoralThesis
http://hdl.handle.net/11584/265953
ita
numberofpages:92
info:eu-repo/semantics/openAccess
Università degli Studi di Cagliari
license:Non specificato