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MEDICAMENT THERAPEUTIQUE POUR LE SYNDROME D'ALPORT
专利权人:
NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY;DAIICHI SANKYO COMPANY, LIMITED
发明人:
IIJIMA, KAZUMOTO,NOZU, KANDAI,SHONO, AKEMI,KOIZUMI, MAKOTO,ONISHI, YOSHIYUKI,TAKAISHI, KIYOSUMI,ADACHI, TOMOMI
申请号:
CA3047894
公开号:
CA3047894A1
申请日:
2017.12.25
申请国别(地区):
CA
年份:
2018
代理人:
摘要:
The purpose of the present invention is to establish a molecular therapy for Alport's syndrome. Provided are the following: a 15 to 30 base-long oligonucleotide formed of a nucleotide sequence complimentary to cDNA of COL4A5 gene, the oligonucleotide having a truncating mutation in COL4A5 gene of an Alport's syndrome patient, and being able to induce the skipping of an exon where the number of bases is a multiple of 3; a pharmaceutically acceptable salt thereof, or a solvate thereof; and a pharmaceutical drug (therapeutic drug for Alport's syndrome) containing the oligonucleotide, a pharmaceutically acceptable salt thereof, or a solvate thereof.
来源网站:
中国工程科技知识中心
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http://www.ckcest.cn/home/

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