The high-throughput capability of proteomics allows simultaneous examination of numerous proteins and makes a global analysis of proteins in cell, tissue, organ or biofluid possible. This strength of proteomics has been extensively applied to examine altered proteins caused by various diseases. Some of these altered proteins may particularly be important for the disease progression and/or complications. Thus, information on these altered proteins is valuable for better understanding of the pathogenesis and pathophysiology of medical diseases. Additionally, functional analysis of such proteins may lead to identification of novel therapeutic targets and development of new drugs for improving therapeutic outcome as well as for preventing serious complications. This review focuses mainly on applications of renal and urinary proteomics to define novel therapeutic targets in kidney diseases. Several recent studies on various kidney diseases have successfully identified altered renal and urinary proteins, some of which may potentially be the novel therapeutic targets. Urinary proteome profiling has also been applied to biomarker discovery that will be useful for clinical diagnostics, prognosis, prediction of treatment response, and development of personalized medicine. Finally, potential roles of proteomics for drug design and discovery are discussed.