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DISEASE MODEL ANIMAL AND DISEASE THERAPEUTIC AGENT コモンズ

外国特許コード F180009394
整理番号 (NU-665)
掲載日 2018年4月20日
出願国 世界知的所有権機関(WIPO)
国際出願番号 2017JP025273
国際公開番号 WO 2018012497
国際出願日 平成29年7月11日(2017.7.11)
国際公開日 平成30年1月18日(2018.1.18)
優先権データ
  • 特願2016-139308 (2016.7.14) JP
発明の名称 (英語) DISEASE MODEL ANIMAL AND DISEASE THERAPEUTIC AGENT コモンズ
発明の概要(英語) The present invention mainly addresses the problem of providing a novel and specific knockout model animal that presents a symptom of a disease accompanied by a synapse abnormality in the brain, a method for producing same, and a therapeutic agent for said disease. The present invention provides a model animal that presents a symptom of a disease (e.g., frontotemporal lobar degeneration (FTLD)) accompanied by a synapse abnormality, which animal is characterized by being produced by specifically knocking out the FUS gene in the brain, for example, by using the Cre-loxP system and CamK2 promotor, for example and a method for producing same, or a screening method for the therapeutic agent for said disease, in which said model animal is used or the like.
特許請求の範囲(英語) [claim1]
1. In the brain at specific gene knockout FUS characterized in that it is produced by, with abnormal synapses exhibit the symptoms of the disorder in an animal model.
[claim2]
2. The knockout, Cre-loxP CamK2 system is carried out using the promoter, according to claim 1 animal model.
[claim3]
3. Disease with abnormal synapses, frontal lobe degeneration (FTLD) or with a motor neuron disease, according to claim 1 or 2 animal model.
[claim4]
4. Animal is a mouse, to any one of claims 1-3 animal model.
[claim5]
5. In inbred animals, any one of claims 1-4 of animal model.
[claim6]
6. Any one of claims 1-5 obtained from an animal model to model the neuronal cell line model or nerve cells.
[claim7]
7. In the brain at specific gene knockout FUS having the number of operation steps characterized in that, the abnormal synapse with the method of manufacturing the animal model exhibits symptoms of the disease.
[claim8]
8. In the brain at specific gene knockout FUS is the number of operation steps, and a promoter CamK2 Cre-loxP is to use the system, the manufacturing method according to claim 7.
[claim9]
9. Disease with abnormal synapses, frontal lobe degeneration (FTLD) or with a motor neuron disease, the manufacturing method according to claim 8 or claim 7.
[claim10]
10. Animal is a mouse, according to any one of claims 7-9 lactonized.
[claim11]
11. In inbred animals, any one of claims 7-10 lactonized.
[claim12]
12. Any one of claims 1-5 of animal model, or according to claim 6 model cells or neuronal cell line model and including the step of using, with the abnormal synapse ulceratives searching for treatment of a disease screening method.
[claim13]
13. Abnormal synapses with disease, frontal lobe degeneration (FTLD) or with a motor neuron disease, screening method according to claim 12.
[claim14]
14. By application of a drug target SynGAPα2 having the step of measuring an increase or decrease characterized in that, the treatment of disorders involving abnormal synapses in order to search for a method of screening for an agent.
[claim15]
15. The disease with abnormal synapses, frontal lobe degeneration (FTLD) or with a motor neuron disease, according to claim 14 screening method.
[claim16]
16. SynGAPα2 or SynGAPα2 of the gene encoding the protein, or drug which enhances the expression of SynGAPα2 in the brain at the SynGAPα2 protein or drug can increase and as an active ingredient, the treatment of disorders involving abnormal synapse agent.
[claim17]
17. The disease with dysregulated abnormality synapses, frontal lobe degeneration (FTLD) or with a motor neuron disease, a therapeutic agent according to claim 16.
[claim18]
18. SynGAPα2 refilled or increase brain characterized in comprising a step, the treatment of disorders involving abnormal synapse method.
[claim19]
19. Abnormal synapses with disease, frontal lobe degeneration (FTLD) or with a motor neuron disease, a method for the treatment according to claim 18.
  • 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
  • NAGOYA UNIVERSITY
  • 発明者(英語)
  • SOBUE GEN
  • ISHIGAKI SHINSUKE
  • YOKOI SATOSHI
国際特許分類(IPC)
指定国 (WO201812497)
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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