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1. (WO2015119989) PROGRANULIN (PGRN) AND ITS DERIVATIVES FOR DIAGNOSIS AND TREATMENT OF LYSOSOMAL STORAGE DISEASES

Pub. No.:    WO/2015/119989    International Application No.:    PCT/US2015/014364
Publication Date: Fri Aug 14 01:59:59 CEST 2015 International Filing Date: Thu Feb 05 00:59:59 CET 2015
IPC: A61P 3/00
Applicants: NEW YORK UNIVERSITY
LIU, Chuanju
JIAN, Jinlong
Inventors: LIU, Chuanju
JIAN, Jinlong
Title: PROGRANULIN (PGRN) AND ITS DERIVATIVES FOR DIAGNOSIS AND TREATMENT OF LYSOSOMAL STORAGE DISEASES
Abstract:
The present invention provides composition and methods for diagnosis and treatment of lysosomal storage diseases and their diagnosis and treatment, including Gaucher' s Disease and Tay-Sachsdisease, and particularly which utilize progranulin (PGRN), or active PGRN peptides, including atsttrin. The invention also provides animal models of lysosomal storage diseases, including Gaucher' s Disease and Tay-Sachsdisease, based on or including PGRN mutations including PGRN null mutants and PGRN gene knock outs.