By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Fresh data on Biotechnology are presented in a new report. According to news reporting from Krakow, Poland, by NewsRx journalists, research stated, "Since 1990 when the first clinical gene therapy trial was conducted, much attention and considerable promise have been given to this form of treatment. Gene therapy has been used with success in patients suffering from severe combined immunodeficiency syndromes (X-SCID and ADA-deficiency), Leber's congenital amaurosis, hemophilia, beta-thalassemia and adrenoleukodystrophy."
The news correspondents obtained a quote from the research from Jagiellonian University, "Last year, the first therapeutic vector (Glybera) for treatment of lipoprotein lipase deficiency has been registered in the European Union. Nevertheless, there are still several numerous issues that need to be improved to make this technique more safe, effective and easily accessible for patients. Introduction of the therapeutic gene to the given cells should provide the level of expression which will restore the production of therapeutic protein to normal values or will provide therapeutic efficacy despite not fully physiological expression. However, in numerous diseases the expression of therapeutic genes has to be kept at certain level for some time, and then might be required to be switched off to be activated again when worsening of the symptoms may aggravate the risk of disease relapse. In such cases the promoters which are regulated by local conditions may be more required. In this article the special emphasis is to discuss the strategies of regulation of gene expression by endogenous stimuli. Particularly, the hypoxia- or miRNA-regulated vectors offer the possibilities of tight but, at the same time, condition-dependent and cell-specific expression. Such means have been already tested in certain pathophysiological conditions."
According to the news reporters, the research concluded: "This creates the chance for the translational approaches required for development of effective treatments of so far incurable diseases."
For more information on this research see: Gene therapy on demand: Site specific regulation of gene therapy. Gene, 2013;525(2):229-238. Gene can be contacted at: Elsevier Science Bv, PO Box 211, 1000 Ae Amsterdam, Netherlands. (Elsevier - www.elsevier.com; Gene - www.elsevier.com/wps/product/cws_home/506033)
Our news journalists report that additional information may be obtained by contacting A. Jazwa, Jagiellonian University, Fac Biochem Biophys & Biotechnol, Dept. of Med Biotechnol, PL-30387 Krakow, Poland. Additional authors for this research include U. Florczyk, A. Jozkowicz and J. Dulak (see also technology.html">Biotechnology).
Keywords for this news article include: Biotechnology, Krakow, Poland, Europe, Treatment, Gene Therapy, Bioengineering
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