Title:
PROPHYLACTIC OR THERAPEUTIC AGENT FOR CYSTIC FIBROSIS
Document Type and Number:
WIPO Patent Application WO/2018/168777
Kind Code:
A1
Abstract:
Provided are a method whereby cystic fibrosis can be prevented or treated by inhibiting a mechanism in which ΔF508 CFTR expressed in the plasma membrane is ubiquitinated by a plasma membrane quality control system and then subjected to endocytosis followed by lysosomal degradation, etc. More specifically, provided are: a prophylactic or therapeutic agent for cystic fibrosis which comprises a specific polypeptide or nucleic acid; and a method for screening a prophylactic or therapeutic agent for cystic fibrosis, said method comprising the following step (1) or (2). (1) A step for measuring an interaction between CFTR having a mutation of lacking phenylalanine at the 508-position or a peptide fragment containing the aforesaid mutation and RFFL or a peptide fragment containing a fragment thereof. (2) A step for measuring the ubiquitin ligase activity of RFFL.
Inventors:
OKIYONEDA TSUKASA (JP)
Application Number:
PCT/JP2018/009524
Publication Date:
September 20, 2018
Filing Date:
March 12, 2018
Export Citation:
Assignee:
KWANSEI GAKUIN EDUCATIONAL FOUND (JP)
International Classes:
G01N33/15; A61K35/76; A61K38/16; A61K39/395; A61P11/00; C12Q1/25; G01N33/50
Foreign References:
| US20160194644A1 | 2016-07-07 | |||
| JP2013545752A | 2013-12-26 | |||
| JP2005525790A | 2005-09-02 | |||
| US20100222408A1 | 2010-09-02 | |||
| JP2014532417A | 2014-12-08 | |||
| JP2011528908A | 2011-12-01 | |||
| JP2002537793A | 2002-11-12 |
Other References:
OKIYONEDA, TSUKASA: "Drug-creation research for obstructive lung disease, in which ubiquitination is targeted", RESEARCH REPORTS OF THE UEHARA MEMORIAL FOUNDATION, vol. 29, 9 December 2015 (2015-12-09), pages 57
OKIYONEDA, TSUKASA: "Peripheral quality control mechanism eliminating aberrant proteins from the plasma membrane", MATHEWS VAN HOLDE APPLING ANTHONY-CAHILL, vol. 86, no. 1, 25 February 2014 (2014-02-25), pages 54 - 58
OKIYONEDA, TSUKASA: "Classification of CFTR collector for the purpose of pharmacotherapy of cystic fibrosis, and synergistic effects", ANNUAL ABSTRACTS OF THE PHARMACEUTICAL SOCIEITY OF JAPAN, vol. 134, 2014, pages 55 - 5
OKIYONEDA, TSUKASA: "Chaperone-Independent Peripheral Quality Control of CFTR by RFFL E3 Ligase", DEVELOPMENTAL CELL, vol. 44, no. 6, 1 March 2018 (2018-03-01), pages 694 - 708, XP055545789
OKIYONEDA, TSUKASA: "Peripheral quality control mechanism eliminating aberrant proteins from the plasma membrane", MATHEWS VAN HOLDE APPLING ANTHONY-CAHILL, vol. 86, no. 1, 25 February 2014 (2014-02-25), pages 54 - 58
OKIYONEDA, TSUKASA: "Classification of CFTR collector for the purpose of pharmacotherapy of cystic fibrosis, and synergistic effects", ANNUAL ABSTRACTS OF THE PHARMACEUTICAL SOCIEITY OF JAPAN, vol. 134, 2014, pages 55 - 5
OKIYONEDA, TSUKASA: "Chaperone-Independent Peripheral Quality Control of CFTR by RFFL E3 Ligase", DEVELOPMENTAL CELL, vol. 44, no. 6, 1 March 2018 (2018-03-01), pages 694 - 708, XP055545789
Attorney, Agent or Firm:
SAEGUSA & PARTNERS (JP)
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