Author/Editor		Ravnik-Glavač, Metka; Glavač, Damjan
Title		Gene therapy of cystic fibrosis: CFTR, vectors and animal models
Type		članek
Source		Period Biol
Vol. and No.		Letnik 100, št. 3
Publication year		1998
Volume		str. 315-8
Language		eng
Abstract		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 these studies 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 gene transfer. 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.
Descriptors		CYSTIC FIBROSIS GENE THERAPY CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR LUNG DISEASES EPITHELIUM GENE TRANSFER ADENOVIRIDAE DEPENDOVIRUS RETROVIRIDAE DISEASE MODELS, ANIMAL