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THERAPEUTIC AGENT FOR NEUROPATHY IN ORGANIC ACIDEMIA OF WHICH MECHANISM RELIES ON INCREASE IN CAMP

Foreign code F180009462
File No. (S2017-0043-N0)
Posted date Jul 31, 2018
Country WIPO
International application number 2017JP037507
International publication number WO 2018074463
Date of international filing Oct 17, 2017
Date of international publication Apr 26, 2018
Priority data
  • P2016-203955 (Oct 17, 2016) JP
Title THERAPEUTIC AGENT FOR NEUROPATHY IN ORGANIC ACIDEMIA OF WHICH MECHANISM RELIES ON INCREASE IN CAMP
Abstract [Problem] To elucidate a mechanism associated with neuropathy in methylmalonic acidemia, and to develop a novel therapeutic agent or the like for neuropathy in organic acidemia on the basis of this finding. [Solution] The present inventors have established techniques for the establishment of iPS cells from a methylmalonic acidemia patient, for the establishment of a culturing method for stably maintaining the iPS cells, and for the differentiation of iPS cells from a methylmalonic acidemia patient into nerve cells, by using peripheral blood lymphocytes from a methylmalonic acidemia patient. It is found that neuropathy in organic acidemia can be treated and prevented by replenishing cAMP by employing the series of experimental techniques. A medicine according to the present invention is characterized by being capable of increasing cAMP in organic acidemia to treat or prevent neuropathy.
Scope of claims [claim1]
1. In viremia organic acid, thereby increasing cAMP, preventing a neurological disorder and not a medicament for the treatment of neurological disorders
[claim2]
2. Further, CREB activating according to claim 1 characterized in that the medicament for the treatment of neurological disorders
[claim3]
3. The organic acid viremia, Adnylate cyclase activity of a therapeutic agent according to claim 1 or 2 Neuronopathies
[claim4]
4. The organic acid viremia, viremia methylmalonic acid, propionic acid viremia selected from any one or more of a therapeutic agent according to claim 1 or 2 Neuronopathies
[claim5]
5. CAMP, Forskolin,GW9508,NECA,SKF77434,nicotinamide, dobutamine,db-cAMP increase made by any one of 4 and any claim 1 from a medicament for the treatment of neurological disorder
[claim6]
6. Increased cAMP, Adenylate cyclase to suppress or prevent a loss in the activity of which is performed by the characterized in that the therapeutic agent according to claim 3 Neuronopathies
[claim7]
7. The prevention and treatment of neurological disorders by not, and to increase the survival benefit from any one of claims 6 claim 1 medicament for the treatment of neurological disorder
[claim8]
8. Using a medicament for the treatment according to any one of claims 1-7 Neuronopathies, viremia neurological disorder in an organic acid and not ETI.HI
  • Applicant
  • ※All designated countries except for US in the data before July 2012
  • KUMAMOTO UNIVERSITY
  • Inventor
  • MATSUMOTO SHIRO
  • ERA TAKUMI
  • ENDO FUMIO
IPC(International Patent Classification)
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