Funding for this research was provided by:
Japan Society for the Promotion of Science (18K07414, 19K12202)
Japan Agency for Medical Research and Development (19ek0109231s0203, 19ek0109231s0103, JP19ek0109231h0003, 19ek0109231h0003)
Ministry of Education, Culture, Sports, Science and Technology (17H04189, 19K08726)
Received: 5 May 2019
Accepted: 12 May 2020
First Online: 2 June 2020
: This study was supported by Grants-in-Aid for Scientific Research (KAKENHI) from the Ministry of Education, Culture, Sports, Science, and Technology of Japan (subject ID: 16K19642 to T.Y. and 26293203, 17H04189 to K.I., and 19K08726 to K.N.), by the Japan Agency for Medical Research and Development (AMED) (Grant nos. JP19ek0109231h0003 to K.N. and K.I., 19ek0109231s0103 to H.K., and 19ek0109231s0203 to M.T.) and Grants-in-Aid from the MEXT/JSPS Grants-in-Aid for Scientific Research (C) (Grant nos. 18K07414 to Y.T. and 19K12202 to A.S.). K.I., K.N., A.S., M.K., Y.O., K.T., and T.A. have filed a patent application on the development of antisense nucleotides for exon skipping therapy of Alport syndrome. This research used computational resources of the K supercomputer provided by the RIKEN Center for Computational Science through the HPCI System Research project (Project ID: hp180288, Y.T., A.S., and K.N.). M.M. has received consulting fees from Daiichi Sankyo Co., Ltd. K.I. has received grant support from Daiichi Sankyo Co., Ltd., consulting fees from Takeda Pharmaceutical Company and Kyowa Hakko Kirin Co., Ltd., and lecture fees from Chugai Pharmaceutical Co., Ltd., Takeda Pharmaceutical Company and Kyowa Hakko Kirin Co., Ltd. K.N. has received lecture fees from Novartis Pharmaceuticals Corporation and consulting fees from Kyowa Hakko Kirin Co., Ltd. The remaining authors declare no competing interests.
: All procedures were reviewed and approved by the Institutional Review Board of Kobe University School of Medicine (Nos. 301 and 1451). Informed consent was obtained from the patient and his parents to develop cultured urine derived cells from the patient’s urine sample and conduct exon skipping therapy for the cultured cells. All animal experiments were approved by the Committee on Animal Experimentation at Daiichi Sankyo Co., Ltd. and Axcelead Drug Discovery Partners, Inc. Japan. Animals were treated in accordance with the Guide for Animal Experimentation issued from the Science Council of Japan in 2006.