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NOVEL THERAPEUTIC TREATMENT OF PROGRANULIN-DEPENDENT DISEASES
专利权人:
LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN
发明人:
CAPELL, Anja,HAASS, Christian,FELLERER, Katrin
申请号:
EPEP2011/004976
公开号:
WO2012/045451A1
申请日:
2011.10.05
申请国别(地区):
WO
年份:
2012
代理人:
摘要:
The present invention relates an alkalizing agent for use in the treatment, acceleration, or amelioration of a disease and/or the improvement of the outcome of a disease, wherein said disease is a disease which benefits from an enhanced and/or restored progranulin level. Said alkalizing drug finds particularly use in the treatment of wound healing, inflammatory diseases, chronic pain and/or neurodegenerative diseases. It is also envisaged that said disease is caused by haploinsufficiency of the progranulin gene (GRN). The present invention also relates to a pharmaceutical composition, package or kit for use in the treatment, acceleration, amelioration of a disease (or symptoms associated thereto) and/or the improvement of the outcome of a disease, wherein said disease is a disease which benefits from an enhanced and/or restored progranulin level, and wherein said kit or package comprises an alkalizing drug and optionally a package insert or instructions. In a further embodiment, the present invention relates to a method of screening for a pharmaceutically active drug for use in the treatment, acceleration, amelioration of a disease (or symptoms associated thereto) and/or the improvement of the outcome of a disease, wherein said disease is a disease which benefits from an enhanced and/or restored progranulin level, said method comprising the following steps: (a) contacting a test-compound with a test cell, and (b) evaluating an alteration in the intracellular pH of said test-cell, wherein an increase in the intracellular pH indicates that said test compound is a pharmaceutically active drug for use in the treatment, acceleration, amelioration of a disease (or symptoms associated thereto) and/or the improvement of the outcome of a disease, wherein said disease is a disease which benefits from an enhanced and/or restored progranulin level.La présente invention concerne un agent alcalifiant destiné à lutilisation dans le traitement, laccélération ou lamélioration dune m
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