Therapeutic perspectives for structural and functional abnormalities of cilia
作者: Yong Joon KimJoon Kim
作者单位: 1Graduate School of Medical Science and Engineering, Korea Advanced Institute of Science and Technology (KAIST)
刊名: Cellular and Molecular Life Sciences, 2019, Vol.76 (19), pp.3695-3709
来源数据库: Springer Journal
DOI: 10.1007/s00018-019-03158-6
关键词: Primary ciliaCiliopathyGenetic disorderDrug development
英文摘要: Abstract(#br)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...
全文获取路径: Springer  (合作)
影响因子:5.615 (2012)