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PROPHYLACTIC OR THERAPEUTIC AGENT FOR CYSTIC FIBROSIS

外国特許コード F180009483
整理番号 KG0186-WO01,(S2017-0529-N0)
掲載日 2018年9月25日
出願国 世界知的所有権機関(WIPO)
国際出願番号 2018JP009524
国際公開番号 WO 2018168777
国際出願日 平成30年3月12日(2018.3.12)
国際公開日 平成30年9月20日(2018.9.20)
優先権データ
  • 特願2017-047626 (2017.3.13) JP
発明の名称 (英語) PROPHYLACTIC OR THERAPEUTIC AGENT FOR CYSTIC FIBROSIS
発明の概要(英語) 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.
従来技術、競合技術の概要(英語) BACKGROUND ART
Cystic fibrosis (Cystic Fibrosis, CF) a high frequency white races to chronic obstructive pulmonary disease is recessive genetic diseases and the main features. 7 Million people world CF present in the patient, rather than fundamental therapy, many patients death by age 40.CF is caused by chlorine ion channel expression in epithelial cells (cystic fibrosis transmembrane conductance regulator, CFTR) gene mutation.CF is found in the majority of the variant Δ F508 CFTR (CFTR in the amino acid sequence the phenylalanine at position 508 are deleted) is transferred, the translation is normal, in order to cause abnormality in the three-dimensional structure of protein, ubiquitin to undergo quality control mechanism in the endoplasmic reticulum.As a result, the degradation of the proteasome variants Δ F508 CFTR, cannot be expressed in the plasma membrane.
In addition, have been developed as a therapeutic CF CFTR collector, part of the expression and the plasma membrane ion channel function Δ F508 CFTR chlorine is improved, the clinical treatment effect is insufficient.The collector or the like is expressed in the plasma membrane Δ F508 CFTR, the plasma membrane by the quality control mechanism to quickly undergo ubiquitination, after endocytosis, sometimes the lysosomal degradation.
  • 出願人(英語)
  • ※2012年7月以前掲載分については米国以外のすべての指定国
  • KWANSEI GAKUIN EDUCATIONAL FOUNDATION
  • 発明者(英語)
  • OKIYONEDA TSUKASA
国際特許分類(IPC)
指定国 National States: AE AG AL AM AO AT AU AZ BA BB BG BH BN BR BW BY BZ CA CH CL CN CO CR CU CZ DE DJ DK DM DO DZ EC EE EG ES FI GB GD GE GH GM GT HN HR HU ID IL IN IR IS JO JP KE KG KH KN KP KR KW KZ LA LC LK LR LS LU LY MA MD ME MG MK MN MW MX MY MZ NA NG NI NO NZ OM PA PE PG PH PL PT QA RO RS RU RW SA SC SD SE SG SK SL SM ST SV SY TH TJ TM TN TR TT TZ UA UG US UZ VC VN ZA ZM ZW
ARIPO: BW GH GM KE LR LS MW MZ NA RW SD SL SZ TZ UG ZM ZW
EAPO: AM AZ BY KG KZ RU TJ TM
EPO: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
OAPI: BF BJ CF CG CI CM GA GN GQ GW KM ML MR NE SN ST TD TG
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