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Structural and molecular basis of FAN1 defects in promoting Huntington's disease

Pluciennik, A.; Li, F.; Phadte, A. A.; Bhatia, M.; Barndt, S.; Monte Carlo, A. R.; Hou, C.-f. D.; Yang, R.; Strock, S.

2024-10-07 biochemistry
10.1101/2024.10.07.617005 bioRxiv
Show abstract

FAN1 is a DNA dependent nuclease whose proper function is essential for maintaining human health. For example, a genetic variant in FAN1, Arg507 to His hastens onset of Huntingtons disease, a repeat expansion disorder for which there is no cure. How the Arg507His mutation affects FAN1 structure and enzymatic function is unknown. Using cryo-EM and biochemistry, we have discovered that FAN1 arginine 507 is critical for its interaction with PCNA, and mutation of Arg507 to His attenuates assembly of the FAN1-PCNA on a disease-relevant extrahelical DNA extrusions formed within DNA repeats. This mutation concomitantly abolishes PCNA-FAN1-dependent cleavage of such extrusions, underscoring the importance of PCNA to the genome stabilizing function of FAN1. These results unravel the molecular basis for a specific mutation in FAN1 that dramatically hastens the onset of Huntingtons disease.

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