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  • Gene therapy of cystic fibrosis: CFTR, vectors and animal models
    Ravnik-Glavač, Metka ; Glavač, Damjan
    Human gene therapy means the introduction and expression of funcional gebnes in somatic, nonreproductive cells in order to reverse of prevent particular disorder. Gene therapy has been extensively ... studied in cystic fibrosis (CF), one of the most common lethal autosomal recessive genetic disease. The most life-treating clinical manifestations of CF are pulmonary disorders. Gene therapy of CF is therefore focused in airway epithelial cells. Phase I clinical trials involving CF patients have been initated to evaluate various techniques for delivery of a functional gene to the lung. The results of thesestudies have clearly demonstrated that gene transfer and gene therapy is very complex task. We review brifly the complexity in CFTR protein function and heterogeneity of CFTR expression in different lung cell types as well as the advantages and limitations connected with various vectors involved in genetransfer. We also introduce our approach to discover naturally occuring CFTR gene mutations in non-human primates which could produce the most appropriate animal CF model for studying the etiology and therapy of CF.
    Vir: Periodicum biologorum : an interdisciplinary international journal of the Societas Scientiarum Naturalium Croatica established 1885. - ISSN 0031-5362 (Letn. 100, št. 3, 1998, str. 315-318)
    Vrsta gradiva - članek, sestavni del
    Leto - 1998
    Jezik - angleški
    COBISS.SI-ID - 8821465

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