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AssigneeNational University Corporation Tokyo Medical and Dental University
InventorHitoshi OKAZAWA
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To provide a compound which enables treatment or prevention of spinocerebellar ataxia, analyses were carried out based on a screening using a spinocerebellar ataxia type 1 (SCA1) fly model and on the like. As a result, the following proteins ameliorating the pathology of spinocerebellar ataxia were identified: RPA1, PNKP, XRCC3, XRCC4, CCNH, POLE, POLH, and PERI. On the other hand, the following proteins aggravating the pathology were identified: CHK1, LIG3, FEN1, LIG1, ERCC5, XAB2, ERCC2, DMC1, RECQL5, MUS81, EME1, SPO11, and BLM. In addition, it has been revealed that ATXN1, which is a cause of SCA1, binds to RPA1, BRCA1, and BRCA2, and suppresses the activities of these proteins, so that the above-described pathology is caused.
An agent for preventing or treating spinocerebellar ataxia, comprising, as an active ingredient, at least one of the following (a) to (d):
A screening method for a candidate compound for preventing or treating spinocerebellar ataxia, the method comprising the following steps (a) to (c):
A screening method for a candidate compound for preventing or treating spinocerebellar ataxia, the method comprising the following steps (a) and (b):
A screening method for a candidate compound for preventing or treating spinocerebellar ataxia, the method comprising the following steps (a) and (b):
Dependencies are inferred from the claim text.