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[Perspectives in gene therapy of polycystic kidney disease]
M Merta1, M Kohoutová, R Rysavá
1I. interní klinika 1. lékarské fakulty a Vseobecné fakultní nemocnice v Praze, Czech Republic.
Abstract:
An overview concerning principles and development of gene therapy from experimental models to first trials of application gene therapy in clinical medicine is provided. Increased attention is focused on specific methods of gene transfer into kidneys. Renal hereditary diseases represent an important subgroup of the diseases of the kidney, leading non rarely to chronic renal failure; (autosomal dominant) polycystic kidney disease remains to be one of the most frequent and clinically important. Preliminary conditions, including technical tools and pathophysiological insights to be fulfilled, as prerequisites for an application of gene therapy in polycystic kidney disease, are discussed.