INHIBITORS OF FAPP2 AND USES THEREOF

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United States of America Patent

APP PUB NO 20160250221A1
SERIAL NO

14907172

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Abstract

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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.

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Patent Owner(s)

Patent OwnerAddress
FONDAZIONE TELETHONITALY ROME ROMA ROME

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
D'ANGELO, Giovanni Pozzuoli (NA), IT 8 195
DE, MATTEIS Maria Antonietta Pozzuoli (NA), IT 1 1
LA, MONTAGNA Raffaele Pozzuoli (NA), IT 2 1

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