Title:
SHORTENED POLYCYSTIN-1 POLYPEPTIDE AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2024/029593
Kind Code:
A1
Abstract:
The present disclosure includes: a polynucleotide comprising a sequence encoding shortened polycystin-1 (PC1) having a cyst formation-inhibiting activity, in which the shortened PC1 contains a PC1 C-terminal tail (CTT) domain or a part thereof; a shortened PC1 polypeptide encoded by the polynucleotide; a vector carrying the polynucleotide; a pharmaceutical composition for treating PKD1-related diseases, which contains the polynucleotide, the polypeptide or the vector; and others.
Inventors:
NISHIKAWA HIROSHI (JP)
OGURI KOUDAI (JP)
MIZUGUCHI HIROSHI (JP)
HAYASHI YOHEI (JP)
SAKAMAKI WAKAKO (JP)
MINENO KURUMI (JP)
HARADA TAKEO (JP)
OGURI KOUDAI (JP)
MIZUGUCHI HIROSHI (JP)
HAYASHI YOHEI (JP)
SAKAMAKI WAKAKO (JP)
MINENO KURUMI (JP)
HARADA TAKEO (JP)
Application Number:
PCT/JP2023/028405
Publication Date:
February 08, 2024
Filing Date:
August 03, 2023
Export Citation:
Assignee:
OTSUKA PHARMA CO LTD (JP)
International Classes:
C12N15/12; A61K38/17; A61K48/00; A61P13/12; A61P43/00; C12N15/63
Other References:
FOY R. L., CHITALIA V. C., PANCHENKO M. V., ZENG L., LOPEZ D., LEE J. W., RANA S. V., BOLETTA A., QIAN F., TSIOKAS L., PIONTEK K. : "Polycystin-1 regulates the stability and ubiquitination of transcription factor Jade-1", HUMAN MOLECULAR GENETICS, OXFORD UNIVERSITY PRESS, GB, vol. 21, no. 26, 15 December 2012 (2012-12-15), GB , pages 5456 - 5471, XP093135178, ISSN: 0964-6906, DOI: 10.1093/hmg/dds391
SU XUEFENG, WU MAOQING, YAO GANG, EL-JOUNI WASSIM, LUO CHONG, TABARI AZADEH, ZHOU JING: "Regulation of polycystin-1 ciliary trafficking by motifs at its C-terminus and polycystin-2 but not cleavage at GPS site", JOURNAL OF CELL SCIENCE, COMPANY OF BIOLOGISTS LIMITED, CAMBRIDGE, Cambridge , XP093135181, ISSN: 0021-9533, DOI: 10.1242/jcs.160556
PARNELL STEPHEN C., MAGENHEIMER BRENDA S., MASER ROBIN L., ZIEN CHRISTOPHER A., FRISCHAUF ANNA-MARIA, CALVET JAMES P.: "Polycystin-1 Activation of c-Jun N-terminal Kinase and AP-1 Is Mediated by Heterotrimeric G Proteins", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, US, vol. 277, no. 22, 1 May 2002 (2002-05-01), US , pages 19566 - 19572, XP093135183, ISSN: 0021-9258, DOI: 10.1074/jbc.M201875200
ONUCHIC LAURA, PADOVANO VALERIA, SCHENA GIORGIA, RAJENDRAN VANATHY, DONG KE, GRESKO NIKOLAY P., SHI XIAOJIAN, SHEN HONGYING, SOMLO: "The C-terminal tail of polycystin-1 suppresses cystic disease in a mitochondrial enzyme-dependent fashion", BIORXIV, 23 December 2021 (2021-12-23), XP093011046, [retrieved on 20230102], DOI: 10.1101/2021.12.21.473680
SU XUEFENG, WU MAOQING, YAO GANG, EL-JOUNI WASSIM, LUO CHONG, TABARI AZADEH, ZHOU JING: "Regulation of polycystin-1 ciliary trafficking by motifs at its C-terminus and polycystin-2 but not cleavage at GPS site", JOURNAL OF CELL SCIENCE, COMPANY OF BIOLOGISTS LIMITED, CAMBRIDGE, Cambridge , XP093135181, ISSN: 0021-9533, DOI: 10.1242/jcs.160556
PARNELL STEPHEN C., MAGENHEIMER BRENDA S., MASER ROBIN L., ZIEN CHRISTOPHER A., FRISCHAUF ANNA-MARIA, CALVET JAMES P.: "Polycystin-1 Activation of c-Jun N-terminal Kinase and AP-1 Is Mediated by Heterotrimeric G Proteins", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, US, vol. 277, no. 22, 1 May 2002 (2002-05-01), US , pages 19566 - 19572, XP093135183, ISSN: 0021-9258, DOI: 10.1074/jbc.M201875200
ONUCHIC LAURA, PADOVANO VALERIA, SCHENA GIORGIA, RAJENDRAN VANATHY, DONG KE, GRESKO NIKOLAY P., SHI XIAOJIAN, SHEN HONGYING, SOMLO: "The C-terminal tail of polycystin-1 suppresses cystic disease in a mitochondrial enzyme-dependent fashion", BIORXIV, 23 December 2021 (2021-12-23), XP093011046, [retrieved on 20230102], DOI: 10.1101/2021.12.21.473680
Attorney, Agent or Firm:
YAMAO, Norihito et al. (JP)
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