| 发明名称 |
Allosteric chaperones and uses thereof |
| 摘要 |
The present invention relates to an allosteric non-inhibitory chaperone of the lysosomal acid alpha-glucosidase (GAA) for use in the treatment of a pathological condition characterized by a deficiency of the lysosomal acid alpha-glucosidase (GAA), to pharmaceutical composition thereof, to a method for increasing the activity of GAA in a subject and to a method for identifying an allosteric non-inhibitory chaperone for GAA. |
| 申请公布号 |
AU2013273473(B2) |
申请公布日期 |
2016.05.26 |
| 申请号 |
AU20130273473 |
申请日期 |
2013.06.06 |
| 申请人 |
CONSIGLIO NAZIONALE DELLE RICERCHE;FONDAZIONE TELETHON |
发明人 |
PARENTI, GIANCARLO;PORTO, CATERINA;MORACCI, MARCO;FERRARA, MARIA CARMINA;COBUCCI-PONZANO, BEATRICE;ANDRIA, GENEROSO |
| 分类号 |
A61K31/198;A61K31/445;A61K38/47;A61P3/00 |
主分类号 |
A61K31/198 |
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