Abstract
TDP2 encodes a 5′-tyrosyl DNA phosphodiesterase that is required
for the efficient repair of double-strand breaks (DSBs) induced by the
abortive activity of TOP2. To date, only two homozygous variants in
TDP2 have been reported in five patients from three unrelated
pedigrees with spinocerebellar ataxia 23 (SCAR23). By whole-exome
sequencing, we identified a novel TDP2 splice-site variant
(c.636+3_636+6del) in two Italian siblings (aged 39 and 43) showing
progressive ataxia, intellectual disability, speech delay, refractory
seizures, and various physical anomalies. The variant caused exon 5
skipping with consequent nonsense-mediated mRNA decay and defective
repair of TOP2-induced DSBs, as demonstrated by the functional assays
performed on the patients’ fibroblasts. Our findings further support the
pathogenic role of biallelic loss-of-function variants of TDP2 in
SCAR23 pathogenesis. Considering the adult age of our patients and their
extensive follow-up, our study delineates in more detail the clinical
phenotype related to the loss of TDP2 activity.