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A-06 Anti-Polyglutamine Disease Agent

技術優勢

ΓùÅ 17-AAG markedly ameliorated motor phenotypes of the SBMA mouse model without toxicityΓùÅ 17-AGG passes through the blood-brain barrierΓùÅ Hsp90 inhibitors have shown to be effective in animal models of Parkinson disease, stroke and autoimmune encephalomyelitisΓùÅ 17-AAG could be a candidate for a therapeutic approach to a wide range of tauopathies

技術應用

New therapeutic approach for Parkinson Disease, Polyglutamine Disease, SBMA and Alzheimer's Disease.

詳細技術說明

17-Allylamino-17-Demethoxygeldanamycin (17-AAG), one of the Hsp90 inhibitors is in clinical trials as a candidate for a new cancer drug. It is known to be an excellent derivative that has diminished side effects, yet the same drug effectiveness as Geldanamycin (a). Adrogen receptor (AR) is one of the Hsp90 client proteins, and is a pathogenic gene product of spinal and bulbar muscular atrophy (SBMA). 17-AAG strongly inhibits progression of polyglutamine disease in a mouse model by inducing digestion of a defective AR protein in (SBMA) (b).

*Abstract

Researchers at Nagoya University h ave found that 17-Allyl amino-17-Demethoxygeldanamycin(17-AAG) , a potent Hsp 90 inhibitor, has the ability to degrade polyglutamine-expanded mutant AR. 17-AG G could be a effective potent drug for spinal and bulbar muscular atrophy (SBMA) and possibly for other related neurodegenerative diseases.

附加資料

Inventor: YAMADA, Shin-ichi | NIWA, Jyun-ichi | SOBUE, Gen
Priority Number: WO2007108434A1
IPC Current: C12N001500 | A61K00317088 | A61K0031711 | A61K004800 | A61P002100 | A61P002500 | A61P002514 | A61P002516 | A61P002528 | A61P004300 | C12N000510 | C12N001509
Assignee Applicant: National University Corporation Nagoya University
Title: EXPRESSION CONSTRUCT FOR DIGESTING AGGREGATING PROTEIN AND METHOD OF INHIBITING THE AGGREGATION OF AGGREGATING PROTEIN | PRODUIT DE RECOMBINAISON D'EXPRESSION DESTINE A DIGERER UNE PROTEINE AGREGANTE ET PROCEDE DESTINE A INHIBER L'AGREGATION D'UNE PROTEIN
Usefulness: EXPRESSION CONSTRUCT FOR DIGESTING AGGREGATING PROTEIN AND METHOD OF INHIBITING THE AGGREGATION OF AGGREGATING PROTEIN | PRODUIT DE RECOMBINAISON D'EXPRESSION DESTINE A DIGERER UNE PROTEINE AGREGANTE ET PROCEDE DESTINE A INHIBER L'AGREGATION D'UNE PROTEINE AGREGANTE
Summary: For degrading the aggregate forming protein, for suppressing the formation of aggregate formed by aggregate forming protein within a target eukaryotic cell (both claimed), and for treating or preventing the disease associated with aggregate forming protein such as familial amyotrophic lateral sclerosis, spinal and bulbar muscular atrophy (SBMA), Parkinson's disease, and Alzheimer's disease.
Novelty: Novel expression construct capable of degrading aggregate forming protein, and comprising nucleic acid encoding archaeal proteasome operably connected to promoter of eukaryotic cell, useful for treating Alzheimer's disease

主要類別

生物醫學

細分類別

DNA /基因工程

國家/地區

美國