Ciliopathies are a group of hereditary disorders that result from structural or 
functional abnormalities of cilia. Recent intense research efforts have 
uncovered the genetic bases of ciliopathies, and our understanding of the 
assembly and functions of cilia has been improved significantly. Although 
mechanism-specific therapies for ciliopathies have not yet received regulatory 
approval, the use of innovative therapeutic modalities such as oligonucleotide 
therapy, gene replacement therapy, and gene editing in addition to symptomatic 
treatments are expected to provide valid treatment options in the near future. 
Moreover, candidate chemical compounds for developing small molecule drugs to 
treat ciliopathies have been identified. This review introduces the key features 
of cilia and ciliopathies, and summarizes the advances as well as the challenges 
that remain with the development of therapies for treating ciliopathies.