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Cilia-related disease model and use thereof

Ref. No. AJ216
Appl. No. (filing date) PCT/JP2020/028474 (2020/07/22)
Pub. No. (Pub. date) WO2021/15245 (2021/01/28)
Priority claim No. (Priority date)

JP2019-135634(2019/07/23)

Patent No.

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Inventor OSAFUNE Kenji, etc.
Assignee Kyoto University
Title of Invention Cilia-related disease model and use thereof
Abstract

The invention relates to a renal cyst-forming model obtained from a renal organoid induced from an iPS cell derived from a patient with a cilia-related disease such as autosomal dominant polycystic kidney (ADPKD), and a method for screening or evaluating a therapeutic agent for cilia-related diseases using the same, wherein the cyst-formation is promoted by culturing in the presence of forskolin or Blebbistatin, etc. The renal cyst-forming model of the invention recapitulates the cilia-related disease phenotype such as cyst formation and decreased calcium release, and thus enables an efficient and highly accurate screening and evaluation of candidate therapeutic agents for cilia-related diseases such as ADPKD by using as indices the morphology of renal cysts and calcium kinetics.

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