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Therapeutic agent for intracellular tdp-43 content-related disease NEW

外国特許コード F170009124
整理番号 (S2016-0233-N0)
掲載日 2017年7月13日
出願国 世界知的所有権機関(WIPO)
国際出願番号 2016JP088738
国際公開番号 WO 2017111166
国際出願日 平成28年12月26日(2016.12.26)
国際公開日 平成29年6月29日(2017.6.29)
優先権データ
  • 特願2015-253332 (2015.12.25) JP
発明の名称 (英語) Therapeutic agent for intracellular tdp-43 content-related disease NEW
発明の概要(英語) (WO2017111166)
According to the present invention, a drug for treating an intracellular TDP-43 content-related disease is developed and provided .
In the binding of TDP-43 to a specific mt-tRNA, the binding sites of the respective substances are identified.
On the basis of the result of the identification, a binding inhibitor capable of selectively inhibiting the binding of TDP-43 to the specific mt-tRNA is developed.
特許請求の範囲(英語) [claim1]
1. The TAR DNA connection protein - being the connection inhibiter of 43 and mt-tRNA,
(A) The molecule which is connected to the territory where it consists of the all or part of the variable territory in mt-tRNA,
(B) The molecule which covers the territory where it consists of the all or part of the variable territory in mt-tRNA,
(C) At the time of amino acid arranging showing with SEQ ID NO 1 the lysine of 136 rank and the lysine of 145 rank, the TAR DNA connection protein which one includes the amino acid which is selected from the group which consists of the phenylalanine of 147 rank and the phenylalanine of 149 rank at least - the molecule which is connected to the territory where it consists of the portion of 43, or
(D) The lysine of 136 rank in amino acid arranging which is shown with SEQ ID NO 1 and the lysine of 145 rank, the TAR DNA connection protein which the amino acid which is selected from the group which consists of the phenylalanine of 147 rank and the phenylalanine of 149 rank includes one at least - the molecule which covers the territory where it consists of the portion of 43,
It consists of,
The description above mt-tRNA, mt-tRNA (Asn), mt-tRNA (Gln), mt-tRNA (Pro) and mt-tRNA (Glu) is each mt-tRNA which is selected from the group which consists of,
The aforementioned connection inhibiter.
[claim2]
2. The aforementioned molecule is formed with nuclear acid, peptide, low-molecular chemical compound, or those combinations, in claim 1 the connection inhibiter of statement.
[claim3]
3. The aforementioned nuclear acid the SEQ ID NO 2 (guguuugugg), the SEQ ID NO 3 (cagggau), the SEQ ID NO 4 (auggugg) and the SEQ ID NO 5 (uuggucg) with sooner or later is shown consists of the nucleotide which includes one base arrangement, the TAR DNA connection protein - connects with 43, in claim 2 the connection inhibiter of statement.
[claim4]
4. It consists of the nucleotide which includes complimentary base arrangement in the territory where it consists of the all or part of the variable territory the aforementioned nuclear acid in the description above mt-tRNA the aforementioned in each case connects to one mt-tRNA, in claim 2 the connection inhibiter of statement.
[claim5]
5. The variable territory in the description above mt-tRNA the SEQ ID NO 6 (cuaaguguuuguggg), the SEQ ID NO 7 (uucucagggauggg), the SEQ ID NO 8 (gcuaaugguggagu), and the SEQ ID NO 9 (aucauuggucgugg) with sooner or later is shown consists of one base arrangement, in claim 4 the connection inhibiter of statement.
[claim6]
6. It includes the amino acid arrangement of 136 rank -149 rank at the time of amino acid arranging which the aforementioned peptide shows with SEQ ID NO 1, consists of the peptide below total length 100 amino acids, the aforementioned in each case it connects to one mt-tRNA, in claim 2 the connection inhibiter of statement.
[claim7]
7. The aforementioned nuclear acid is the gene revelation system which includes the polynucleotide which the peptide of statement the cord/code is done in claim 6, in claim 2 the connection inhibiter of statement.
[claim8]
8. Either of the claim 1-7 in one section the TAR DNA connection protein of statement - the medicine composition for abundance-related diseased remedy inside TAR DNA connection protein - 43 cells which designate the connection inhibiter of 43 and mt-tRNA as the active ingredient.
[claim9]
9. The abundance-related disease inside the aforementioned TAR DNA connection protein - 43 cells the muscle atrophy characteristic lateral column muscardine, the frontal side head leaf denaturation symptom, is selected from the group which consists of the Alzheimer's disease, and the parkinson's disease, in claim 8 the medicine composition of statement.
  • 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
  • TOKYO UNIVERSITY OF AGRICULTURE AND TECHNOLOGY
  • TOKYO METROPOLITAN UNIVERSITY
  • 発明者(英語)
  • IZUMIKAWA KEIICHI
  • TAKAHASHI NOBUHIRO
  • ISHIKAWA HIDEAKI
  • ISOBE TOSHIAKI
国際特許分類(IPC)
指定国 (WO2017111166)
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 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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