Emerging therapeutic targets in paediatric kidney diseases

Abstract

During the past few years our knowledge of the structural and functional molecules of kidney glomeruli and tubuli has rapidly increased. In addition, the regulatory mechanisms of kidney morphogenesis are resolving. These data lay the basis for the efforts towards various targeted therapies of kidney diseases. In experimental models, monoclonal antibodies, antisense technology and targeted gene transfer have given promises of the efficacy of new therapeutic approaches. Transfer of these strategies from bench to bedside is now the major task. However, gene therapy of renal diseases, for instance, is far away from clinical trials. The development of optimal vectors and especially the long-term expression of targeted genes are still major obstacles.

• Alport syndrome
• congenital nephrosis
• inductive tissue interaction
• kidney morphogenesis
• polycystic renal disease
• Wilms tumour