Transcriptome analysis of cep290 knockdown kidney in Xenopus embryos.

Mutations in CEP290 are known to cause renal failure with renal cysts. To investigate the molecular mechanism of renal injury associated with CEP290 mutation, we performed transcriptome analysis of cep290 knockdown kidney in Xenopus embryos. While there is no established treatment for renal disease associated with the CEP290 mutations, Tolvaptan, which is used for the treatment of the autosomal dominant polycystic kidney disease that is the most common disease with renal cyst formation, may be a clinically possible medicine for renal cysts caused by CEP290 mutation. To explore the possibility of drug repositioning in the CEP290 mutated renal cyst formation, we also performed the transcriptome analysis of cep290 knockdown Xenopus kidneys with or without Tolvaptan treatment.