Method for screening compound for treating and / or preventing disease in which aggregates of TDP-43 accumulate

摘要

PROBLEM TO BE SOLVED: To provide TDP-43 variants having agglomerate-forming ability and cytotoxicity in the full length sequence, and methods for screening compounds for treating and/or preventing diseases associated with TDP-43 agglomerate accumulation using the TDP-43 variants.SOLUTION: The invention provides a TDP-43 variant comprising an amino acid sequence where cysteine at position 173 and/or position 175 in the amino acid sequence of TDP-43 is/are substituted. The invention also provides a method for screening a compound for treating and/or preventing a disease associated with TDP-43 agglomerate accumulation, which method comprises the steps of: (1) contacting the TDP-43 variant with a candidate compound; (2) detecting an activity of the compound to inhibit agglomerate formation; and (3) selecting a compound that inhibits the agglomerate formation.SELECTED DRAWING: Figure 6