D'Angelo, Giovanni,De Matteis, Maria Antonietta,La Montagna, Raffaele,DAngelo, Giovanni
申请号：
AU2014295000
公开号：
AU2014295000A1
申请日:
2014.07.25
申请国别(地区):
AU
年份:
2016
代理人:
摘要：
The present invention provides methods and compositions for reducing globotriaosylceramide (Gb3) accumulation and treating diseases, disorders or conditions associated with Gb3 accumulation based on inhibitors of phosphatidylinositol-4-phosphate adaptor-2 (FAPP2), including interfering oligonucleotides, for example, siRNAs, and small molecule compounds based inhibitors. The present invention is particularly useful in treating Fabry disease and other sphingolipidoses relating to sphingolipid metabolism, such as Gaucher's disease.
