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  • Gensko zdravljenje cistične fibroze - napredek in omejitve = Gene therapy of cystic fibrosis - progress and limitations
    Ravnik-Glavač, Metka ; Glavač, Damjan
    Background. Human gene therapy means the introduction and expression of functional genes in somatic, nonreproductive cells in order to reverse or prevent a particular disorder. Cystic fibrosis (CF) ... is one of the most common monogenic diseases of white populations which is incurable with currently usedtherapeutic methods. Because molecular bases of the disease are relativelywell determined, cystic fibrosis has become a model disease in studying gene therapy. We describe here the molecular bases of cystic fibrosis, vectors for transfer of therapeutic genes into airways and phase I clinical trials of human gene therapy. Although a lot of studies have been done and great success has been achieved in the last few years, it is not yet possible to cure diseases with gene therapy. Conclusions. New knowledge has raised new questions concerning the complexity in CFTR protein function and heterogeneity of CFTR expression in different lung cell types as well as the limitations connected with various vectors involved in gene transfer. The future gene therapy will probably be in utero gene therapy of embryos with CF.Its progress greatly depends on the development of appropriate animal models, especially the non-human primate CF model for studying the efficiency of gene therapy. Efficient gene therapy will probably not be possible in the near future. However, whenever the knowledge will turn the tide in favor of CFpatients, it will bring great hope and relief for thousands of young patients and their parents all over the world.
    Vir: Slovenska pediatrija : revija Združenja pediatrov Slovenije in Združenja specialistov šolske in visokošolske medicine Slovenije. - ISSN 1318-4423 (Letn. 7, suppl. 1, 2000, str. 126-131)
    Vrsta gradiva - prispevek na konferenci
    Leto - 2000
    Jezik - slovenski
    COBISS.SI-ID - 11347417

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