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RFC1 regulates the expansion of neural progenitors in the developing zebrafish cerebellum

Nobilleau, F.; Audet, S.; Turk, S.; Zaouter, C.; Liao, M.; Pilon, N.; Tetreault, M.; Patten, S. A.; Samarut, E.

2024-06-28 developmental biology
10.1101/2024.06.28.601164 bioRxiv
Show abstract

DNA replication and repair are basic yet essential molecular processes for all cells. RFC1 encodes the largest subunit of the Replication Factor C (RFC), which is a clamp-loader during DNA replication and repair. Intronic repeat expansion in RFC1 has recently been associated with so-called RFC1-related disorders, which mainly encompass late-onset cerebellar ataxias. However, the mechanisms that make certain tissues more susceptible to defects in these universal pathways remain mysterious. In this study, we provide the first investigation of RFC1 gene function in vivo using zebrafish. We showed that RFC1 is expressed in neural progenitor cells within the developing cerebellum and that it is necessary to maintain these cells genomic integrity during neurogenic maturation. Accordingly, RFC1 loss-of-function leads to a severe cerebellar phenotype due to impaired neurogenesis of both Purkinje and granule cells. Our data thus point to a specific role of RFC1 in the developing cerebellum, paving the way for a better understanding of the pathogenic mechanisms underlying RFC1-related disorders.

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