Inhibitors of FAPP2 and uses thereof

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.