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DNA Polymerase Epsilon Deficiency Causes IMAGe Syndrome with Variable Immunodeficiency.

Published version
Peer-reviewed

Type

Article

Change log

Authors

Logan, Clare V 
Murray, Jennie E 
Parry, David A 
Robertson, Andrea 
Bellelli, Roberto 

Abstract

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.

Description

Keywords

DNA replication, IMAGe syndrome, adrenal failure, cell cycle, growth, immunodeficiency, microcephaly, polymerase epsilon, Adolescent, Adrenal Insufficiency, Adult, Alleles, Child, Child, Preschool, Cyclin-Dependent Kinase Inhibitor p57, DNA Polymerase II, DNA Replication, Female, Fetal Growth Retardation, Humans, Infant, Male, Middle Aged, Mutation, Osteochondrodysplasias, Phenotype, Poly-ADP-Ribose Binding Proteins, Urogenital Abnormalities, Young Adult

Journal Title

Am J Hum Genet

Conference Name

Journal ISSN

0002-9297
1537-6605

Volume Title

103

Publisher

Elsevier BV