[Prospects of gene therapy].
In recent years extensive development of gene therapy strategies has been observed. The studies on gene therapy focus on two different research methods. The first one concerns replacement of damaged genes in somatic cells of the organism by correcting genes. Application of modem methods of gene therapy permits to treat a disease already at the molecular level. Therefore, the second method deals with modification of gene expression or obstruction of pathogenic biosynthesis of proteins. Correct genes are introduced to cells by special vectors. The retroviral and adenoviral vectors are most frequently used clinically but very often the plasmid DNA is also useful. The number of clinical trials has been rapidly increasing. Cancer, monogenic diseases and cardiovascular diseases are the main targets of clinical gene therapy. Many hopes are linked with the gene therapy of autoimmune diseases in particular by blocking expression of inflammatory cytokines or neutralising cytokine's receptors. Recently discovered phenomenon of interference of RNA (RNAi) has initiated research on inhibition of selected gene expression by using small interfering RNA (siRNA). Despite the significant methodological progress, allowing safe gene therapy still represents a challenge and requires further intensive research.
Barańska
Małgorzata
M
Z Zakładu Farmakogenetyki Uniwersytetu Medycznego w Lodzi.
Skretkowicz
Jadwiga
J
D000818:Animals; D005786:Gene Expression Regulation / Q000187:drug effects; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000627:therapeutic use; D006801:Humans; D034741:RNA, Small Interfering / Q000494:pharmacology
D034741:RNA, Small Interfering