Increased RPA1 gene dosage affects genomic stability potentially contributing to 17p13.3 duplication syndrome.

A novel microduplication syndrome involving various-sized contiguous duplications in 17p13.3 has recently been described, suggesting that increased copy number of genes in 17p13.3, particularly PAFAH1B1, is associated with clinical features including facial dysmorphism, developmental delay, and auti...

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Main Authors: Emily Outwin, Gillian Carpenter, Weimin Bi, Marjorie A Withers, James R Lupski, Mark O'Driscoll
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2011-08-01
Series:PLoS Genetics
Online Access:https://journals.plos.org/plosgenetics/article/file?id=10.1371/journal.pgen.1002247&type=printable
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author Emily Outwin
Gillian Carpenter
Weimin Bi
Marjorie A Withers
James R Lupski
Mark O'Driscoll
author_facet Emily Outwin
Gillian Carpenter
Weimin Bi
Marjorie A Withers
James R Lupski
Mark O'Driscoll
author_sort Emily Outwin
collection DOAJ
description A novel microduplication syndrome involving various-sized contiguous duplications in 17p13.3 has recently been described, suggesting that increased copy number of genes in 17p13.3, particularly PAFAH1B1, is associated with clinical features including facial dysmorphism, developmental delay, and autism spectrum disorder. We have previously shown that patient-derived cell lines from individuals with haploinsufficiency of RPA1, a gene within 17p13.3, exhibit an impaired ATR-dependent DNA damage response (DDR). Here, we show that cell lines from patients with duplications specifically incorporating RPA1 exhibit a different although characteristic spectrum of DDR defects including abnormal S phase distribution, attenuated DNA double strand break (DSB)-induced RAD51 chromatin retention, elevated genomic instability, and increased sensitivity to DNA damaging agents. Using controlled conditional over-expression of RPA1 in a human model cell system, we also see attenuated DSB-induced RAD51 chromatin retention. Furthermore, we find that transient over-expression of RPA1 can impact on homologous recombination (HR) pathways following DSB formation, favouring engagement in aberrant forms of recombination and repair. Our data identifies unanticipated defects in the DDR associated with duplications in 17p13.3 in humans involving modest RPA1 over-expression.
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publisher Public Library of Science (PLoS)
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spelling doaj-art-edf5e486d72649e8982bfd132cad59152025-08-20T02:05:36ZengPublic Library of Science (PLoS)PLoS Genetics1553-73901553-74042011-08-0178e100224710.1371/journal.pgen.1002247Increased RPA1 gene dosage affects genomic stability potentially contributing to 17p13.3 duplication syndrome.Emily OutwinGillian CarpenterWeimin BiMarjorie A WithersJames R LupskiMark O'DriscollA novel microduplication syndrome involving various-sized contiguous duplications in 17p13.3 has recently been described, suggesting that increased copy number of genes in 17p13.3, particularly PAFAH1B1, is associated with clinical features including facial dysmorphism, developmental delay, and autism spectrum disorder. We have previously shown that patient-derived cell lines from individuals with haploinsufficiency of RPA1, a gene within 17p13.3, exhibit an impaired ATR-dependent DNA damage response (DDR). Here, we show that cell lines from patients with duplications specifically incorporating RPA1 exhibit a different although characteristic spectrum of DDR defects including abnormal S phase distribution, attenuated DNA double strand break (DSB)-induced RAD51 chromatin retention, elevated genomic instability, and increased sensitivity to DNA damaging agents. Using controlled conditional over-expression of RPA1 in a human model cell system, we also see attenuated DSB-induced RAD51 chromatin retention. Furthermore, we find that transient over-expression of RPA1 can impact on homologous recombination (HR) pathways following DSB formation, favouring engagement in aberrant forms of recombination and repair. Our data identifies unanticipated defects in the DDR associated with duplications in 17p13.3 in humans involving modest RPA1 over-expression.https://journals.plos.org/plosgenetics/article/file?id=10.1371/journal.pgen.1002247&type=printable
spellingShingle Emily Outwin
Gillian Carpenter
Weimin Bi
Marjorie A Withers
James R Lupski
Mark O'Driscoll
Increased RPA1 gene dosage affects genomic stability potentially contributing to 17p13.3 duplication syndrome.
PLoS Genetics
title Increased RPA1 gene dosage affects genomic stability potentially contributing to 17p13.3 duplication syndrome.
title_full Increased RPA1 gene dosage affects genomic stability potentially contributing to 17p13.3 duplication syndrome.
title_fullStr Increased RPA1 gene dosage affects genomic stability potentially contributing to 17p13.3 duplication syndrome.
title_full_unstemmed Increased RPA1 gene dosage affects genomic stability potentially contributing to 17p13.3 duplication syndrome.
title_short Increased RPA1 gene dosage affects genomic stability potentially contributing to 17p13.3 duplication syndrome.
title_sort increased rpa1 gene dosage affects genomic stability potentially contributing to 17p13 3 duplication syndrome
url https://journals.plos.org/plosgenetics/article/file?id=10.1371/journal.pgen.1002247&type=printable
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