[PDF][PDF] DNA polymerase epsilon deficiency causes IMAGe syndrome with variable immunodeficiency

CV Logan, JE Murray, DA Parry, A Robertson… - The American Journal of …, 2018 - cell.com
CV Logan, JE Murray, DA Parry, A Robertson, R Bellelli, Ž Tarnauskaitė, R Challis, L Cleal…
The American Journal of Human Genetics, 2018cell.com
During genome replication, polymerase epsilon (Pol ε) acts as the major leading-strand
DNA polymerase. Here we report the identification of biallelic mutations in POLE, encoding
the Pol ε catalytic subunit POLE1, in 15 individuals from 12 families. Phenotypically, these
individuals had clinical features closely resembling IMAGe syndrome (intrauterine growth
restriction [IUGR], metaphyseal dysplasia, adrenal hypoplasia congenita, and genitourinary
anomalies in males), a disorder previously associated with gain-of-function mutations in …
During genome replication, polymerase epsilon (Pol ε) acts as the major leading-strand DNA polymerase. Here we report the identification of biallelic mutations in POLE, encoding the Pol ε catalytic subunit POLE1, in 15 individuals from 12 families. Phenotypically, these individuals had clinical features closely resembling IMAGe syndrome (intrauterine growth restriction [IUGR], metaphyseal dysplasia, adrenal hypoplasia congenita, and genitourinary anomalies in males), a disorder previously associated with gain-of-function mutations in CDKN1C. POLE1-deficient individuals also exhibited distinctive facial features and variable immune dysfunction with evidence of lymphocyte deficiency. All subjects shared the same intronic variant (c.1686+32C>G) as part of a common haplotype, in combination with different loss-of-function variants in trans. The intronic variant alters splicing, and together the biallelic mutations lead to cellular deficiency of Pol ε and delayed S-phase progression. In summary, we establish POLE as a second gene in which mutations cause IMAGe syndrome. These findings add to a growing list of disorders due to mutations in DNA replication genes that manifest growth restriction alongside adrenal dysfunction and/or immunodeficiency, consolidating these as replisome phenotypes and highlighting a need for future studies to understand the tissue-specific development roles of the encoded proteins.
cell.com