Distinct genetic architectures for syndromic and nonsyndromic congenital heart defects identified by exome sequencing

Congenital heart defects (CHDs) have a neonatal incidence of 0.8–1%. Despite abundant examples of monogenic CHD in humans and mice, CHD has a low absolute sibling recurrence risk (~2.7%), suggesting a considerable role for de novo mutations (DNMs) and/or incomplete penetrance. De novo protein-trunca...

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Main Authors: Sifrim, Alejandro, Hitz, Marc-Phillip, Wilsdon, Anna, Breckpot, Jeroen, Turki, Saeed H Al, Thienpont, Bernard, McRae, Jeremy, Fitzgerald, Tomas W, Singh, Tarjinder, Swaminathan, Ganesh Jawahar, Prigmore, Elena, Rajan, Diana, Abdul-Khaliq, Hashim, Banka, Siddharth, Bauer, Ulrike M.M., Bentham, Jamie, Berger, Felix, Bhattacharya, Shoumo, Bu'Lock, Frances A., Canham, Natalie, Colgiu, Irina-Gabriela, Cosgrove, Catherine, Cox, Helen, Daehnert, Ingo, Daly, Allan, Danesh, John, Fryer, Alan, Gewillig, Marc, Hobson, Emma, Hoff, Kirstin, Homfray, Tessa, Kahlert, Anne-Karin, Ketley, Ami, Kramer, Hans-Heiner, Lachlan, Katherine, Lampe, Anne Katrin, Louw, Jacoba J., Manickara, Ashok Kumar, Manase, Dorin, McCarthy, Karen P., Metcalfe, Kay, Moore, Carmel, Newbury-Ecob, Ruth, Omer, Seham Osman, Ouwehand, Willem H., Park, Soo-Mi, Parker, Michael J., Pickardt, Thomas, Pollard, Martin O., Robert, Leema, Roberts, David J., Sambrook, Jennifer, Setchfield, Kerry, Stiller, Brigitte, Thornborough, Christopher, Toka, Okan, Watkins, Hugh, Williams, Denise, Wright, Michael, Mital, Seema, Daubeney, Piers E.F., Keavney, Bernard, Goodship, Judith, Abu-Sulaiman, Riyadh Mahdi, Klaassen, Sabine, Wright, Caroline F., Firth, Helen V., Barrett, Jeffrey C., Devriendt, Koenraad, FitzPatrick, David R., Brook, J. David, Hurles, Matthew E.
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Published: Nature Publishing Group 2016
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Online Access:https://eprints.nottingham.ac.uk/38584/
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author Sifrim, Alejandro
Hitz, Marc-Phillip
Wilsdon, Anna
Breckpot, Jeroen
Turki, Saeed H Al
Thienpont, Bernard
McRae, Jeremy
Fitzgerald, Tomas W
Singh, Tarjinder
Swaminathan, Ganesh Jawahar
Prigmore, Elena
Rajan, Diana
Abdul-Khaliq, Hashim
Banka, Siddharth
Bauer, Ulrike M.M.
Bentham, Jamie
Berger, Felix
Bhattacharya, Shoumo
Bu'Lock, Frances A.
Canham, Natalie
Colgiu, Irina-Gabriela
Cosgrove, Catherine
Cox, Helen
Daehnert, Ingo
Daly, Allan
Danesh, John
Fryer, Alan
Gewillig, Marc
Hobson, Emma
Hoff, Kirstin
Homfray, Tessa
Kahlert, Anne-Karin
Ketley, Ami
Kramer, Hans-Heiner
Lachlan, Katherine
Lampe, Anne Katrin
Louw, Jacoba J.
Manickara, Ashok Kumar
Manase, Dorin
McCarthy, Karen P.
Metcalfe, Kay
Moore, Carmel
Newbury-Ecob, Ruth
Omer, Seham Osman
Ouwehand, Willem H.
Park, Soo-Mi
Parker, Michael J.
Pickardt, Thomas
Pollard, Martin O.
Robert, Leema
Roberts, David J.
Sambrook, Jennifer
Setchfield, Kerry
Stiller, Brigitte
Thornborough, Christopher
Toka, Okan
Watkins, Hugh
Williams, Denise
Wright, Michael
Mital, Seema
Daubeney, Piers E.F.
Keavney, Bernard
Goodship, Judith
Abu-Sulaiman, Riyadh Mahdi
Klaassen, Sabine
Wright, Caroline F.
Firth, Helen V.
Barrett, Jeffrey C.
Devriendt, Koenraad
FitzPatrick, David R.
Brook, J. David
Hurles, Matthew E.
author_facet Sifrim, Alejandro
Hitz, Marc-Phillip
Wilsdon, Anna
Breckpot, Jeroen
Turki, Saeed H Al
Thienpont, Bernard
McRae, Jeremy
Fitzgerald, Tomas W
Singh, Tarjinder
Swaminathan, Ganesh Jawahar
Prigmore, Elena
Rajan, Diana
Abdul-Khaliq, Hashim
Banka, Siddharth
Bauer, Ulrike M.M.
Bentham, Jamie
Berger, Felix
Bhattacharya, Shoumo
Bu'Lock, Frances A.
Canham, Natalie
Colgiu, Irina-Gabriela
Cosgrove, Catherine
Cox, Helen
Daehnert, Ingo
Daly, Allan
Danesh, John
Fryer, Alan
Gewillig, Marc
Hobson, Emma
Hoff, Kirstin
Homfray, Tessa
Kahlert, Anne-Karin
Ketley, Ami
Kramer, Hans-Heiner
Lachlan, Katherine
Lampe, Anne Katrin
Louw, Jacoba J.
Manickara, Ashok Kumar
Manase, Dorin
McCarthy, Karen P.
Metcalfe, Kay
Moore, Carmel
Newbury-Ecob, Ruth
Omer, Seham Osman
Ouwehand, Willem H.
Park, Soo-Mi
Parker, Michael J.
Pickardt, Thomas
Pollard, Martin O.
Robert, Leema
Roberts, David J.
Sambrook, Jennifer
Setchfield, Kerry
Stiller, Brigitte
Thornborough, Christopher
Toka, Okan
Watkins, Hugh
Williams, Denise
Wright, Michael
Mital, Seema
Daubeney, Piers E.F.
Keavney, Bernard
Goodship, Judith
Abu-Sulaiman, Riyadh Mahdi
Klaassen, Sabine
Wright, Caroline F.
Firth, Helen V.
Barrett, Jeffrey C.
Devriendt, Koenraad
FitzPatrick, David R.
Brook, J. David
Hurles, Matthew E.
author_sort Sifrim, Alejandro
building Nottingham Research Data Repository
collection Online Access
description Congenital heart defects (CHDs) have a neonatal incidence of 0.8–1%. Despite abundant examples of monogenic CHD in humans and mice, CHD has a low absolute sibling recurrence risk (~2.7%), suggesting a considerable role for de novo mutations (DNMs) and/or incomplete penetrance. De novo protein-truncating variants (PTVs) have been shown to be enriched among the 10% of 'syndromic' patients with extra-cardiac manifestations. We exome sequenced 1,891 probands, including both syndromic CHD (S-CHD, n = 610) and nonsyndromic CHD (NS-CHD, n = 1,281). In S-CHD, we confirmed a significant enrichment of de novo PTVs but not inherited PTVs in known CHD-associated genes, consistent with recent findings. Conversely, in NS-CHD we observed significant enrichment of PTVs inherited from unaffected parents in CHD-associated genes. We identified three genome-wide significant S-CHD disorders caused by DNMs in CHD4, CDK13 and PRKD1. Our study finds evidence for distinct genetic architectures underlying the low sibling recurrence risk in S-CHD and NS-CHD.
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spelling nottingham-385842020-05-04T17:58:41Z https://eprints.nottingham.ac.uk/38584/ Distinct genetic architectures for syndromic and nonsyndromic congenital heart defects identified by exome sequencing Sifrim, Alejandro Hitz, Marc-Phillip Wilsdon, Anna Breckpot, Jeroen Turki, Saeed H Al Thienpont, Bernard McRae, Jeremy Fitzgerald, Tomas W Singh, Tarjinder Swaminathan, Ganesh Jawahar Prigmore, Elena Rajan, Diana Abdul-Khaliq, Hashim Banka, Siddharth Bauer, Ulrike M.M. Bentham, Jamie Berger, Felix Bhattacharya, Shoumo Bu'Lock, Frances A. Canham, Natalie Colgiu, Irina-Gabriela Cosgrove, Catherine Cox, Helen Daehnert, Ingo Daly, Allan Danesh, John Fryer, Alan Gewillig, Marc Hobson, Emma Hoff, Kirstin Homfray, Tessa Kahlert, Anne-Karin Ketley, Ami Kramer, Hans-Heiner Lachlan, Katherine Lampe, Anne Katrin Louw, Jacoba J. Manickara, Ashok Kumar Manase, Dorin McCarthy, Karen P. Metcalfe, Kay Moore, Carmel Newbury-Ecob, Ruth Omer, Seham Osman Ouwehand, Willem H. Park, Soo-Mi Parker, Michael J. Pickardt, Thomas Pollard, Martin O. Robert, Leema Roberts, David J. Sambrook, Jennifer Setchfield, Kerry Stiller, Brigitte Thornborough, Christopher Toka, Okan Watkins, Hugh Williams, Denise Wright, Michael Mital, Seema Daubeney, Piers E.F. Keavney, Bernard Goodship, Judith Abu-Sulaiman, Riyadh Mahdi Klaassen, Sabine Wright, Caroline F. Firth, Helen V. Barrett, Jeffrey C. Devriendt, Koenraad FitzPatrick, David R. Brook, J. David Hurles, Matthew E. Congenital heart defects (CHDs) have a neonatal incidence of 0.8–1%. Despite abundant examples of monogenic CHD in humans and mice, CHD has a low absolute sibling recurrence risk (~2.7%), suggesting a considerable role for de novo mutations (DNMs) and/or incomplete penetrance. De novo protein-truncating variants (PTVs) have been shown to be enriched among the 10% of 'syndromic' patients with extra-cardiac manifestations. We exome sequenced 1,891 probands, including both syndromic CHD (S-CHD, n = 610) and nonsyndromic CHD (NS-CHD, n = 1,281). In S-CHD, we confirmed a significant enrichment of de novo PTVs but not inherited PTVs in known CHD-associated genes, consistent with recent findings. Conversely, in NS-CHD we observed significant enrichment of PTVs inherited from unaffected parents in CHD-associated genes. We identified three genome-wide significant S-CHD disorders caused by DNMs in CHD4, CDK13 and PRKD1. Our study finds evidence for distinct genetic architectures underlying the low sibling recurrence risk in S-CHD and NS-CHD. Nature Publishing Group 2016-08-01 Article PeerReviewed Sifrim, Alejandro, Hitz, Marc-Phillip, Wilsdon, Anna, Breckpot, Jeroen, Turki, Saeed H Al, Thienpont, Bernard, McRae, Jeremy, Fitzgerald, Tomas W, Singh, Tarjinder, Swaminathan, Ganesh Jawahar, Prigmore, Elena, Rajan, Diana, Abdul-Khaliq, Hashim, Banka, Siddharth, Bauer, Ulrike M.M., Bentham, Jamie, Berger, Felix, Bhattacharya, Shoumo, Bu'Lock, Frances A., Canham, Natalie, Colgiu, Irina-Gabriela, Cosgrove, Catherine, Cox, Helen, Daehnert, Ingo, Daly, Allan, Danesh, John, Fryer, Alan, Gewillig, Marc, Hobson, Emma, Hoff, Kirstin, Homfray, Tessa, Kahlert, Anne-Karin, Ketley, Ami, Kramer, Hans-Heiner, Lachlan, Katherine, Lampe, Anne Katrin, Louw, Jacoba J., Manickara, Ashok Kumar, Manase, Dorin, McCarthy, Karen P., Metcalfe, Kay, Moore, Carmel, Newbury-Ecob, Ruth, Omer, Seham Osman, Ouwehand, Willem H., Park, Soo-Mi, Parker, Michael J., Pickardt, Thomas, Pollard, Martin O., Robert, Leema, Roberts, David J., Sambrook, Jennifer, Setchfield, Kerry, Stiller, Brigitte, Thornborough, Christopher, Toka, Okan, Watkins, Hugh, Williams, Denise, Wright, Michael, Mital, Seema, Daubeney, Piers E.F., Keavney, Bernard, Goodship, Judith, Abu-Sulaiman, Riyadh Mahdi, Klaassen, Sabine, Wright, Caroline F., Firth, Helen V., Barrett, Jeffrey C., Devriendt, Koenraad, FitzPatrick, David R., Brook, J. David and Hurles, Matthew E. (2016) Distinct genetic architectures for syndromic and nonsyndromic congenital heart defects identified by exome sequencing. Nature Genetics, 48 (9). pp. 1060-1065. ISSN 1546-1718 Clinical Genetics Congenital Heart Defects Genetics Research http://www.nature.com/ng/journal/v48/n9/full/ng.3627.html doi:10.1038/ng.3627 doi:10.1038/ng.3627
spellingShingle Clinical Genetics
Congenital Heart Defects
Genetics Research
Sifrim, Alejandro
Hitz, Marc-Phillip
Wilsdon, Anna
Breckpot, Jeroen
Turki, Saeed H Al
Thienpont, Bernard
McRae, Jeremy
Fitzgerald, Tomas W
Singh, Tarjinder
Swaminathan, Ganesh Jawahar
Prigmore, Elena
Rajan, Diana
Abdul-Khaliq, Hashim
Banka, Siddharth
Bauer, Ulrike M.M.
Bentham, Jamie
Berger, Felix
Bhattacharya, Shoumo
Bu'Lock, Frances A.
Canham, Natalie
Colgiu, Irina-Gabriela
Cosgrove, Catherine
Cox, Helen
Daehnert, Ingo
Daly, Allan
Danesh, John
Fryer, Alan
Gewillig, Marc
Hobson, Emma
Hoff, Kirstin
Homfray, Tessa
Kahlert, Anne-Karin
Ketley, Ami
Kramer, Hans-Heiner
Lachlan, Katherine
Lampe, Anne Katrin
Louw, Jacoba J.
Manickara, Ashok Kumar
Manase, Dorin
McCarthy, Karen P.
Metcalfe, Kay
Moore, Carmel
Newbury-Ecob, Ruth
Omer, Seham Osman
Ouwehand, Willem H.
Park, Soo-Mi
Parker, Michael J.
Pickardt, Thomas
Pollard, Martin O.
Robert, Leema
Roberts, David J.
Sambrook, Jennifer
Setchfield, Kerry
Stiller, Brigitte
Thornborough, Christopher
Toka, Okan
Watkins, Hugh
Williams, Denise
Wright, Michael
Mital, Seema
Daubeney, Piers E.F.
Keavney, Bernard
Goodship, Judith
Abu-Sulaiman, Riyadh Mahdi
Klaassen, Sabine
Wright, Caroline F.
Firth, Helen V.
Barrett, Jeffrey C.
Devriendt, Koenraad
FitzPatrick, David R.
Brook, J. David
Hurles, Matthew E.
Distinct genetic architectures for syndromic and nonsyndromic congenital heart defects identified by exome sequencing
title Distinct genetic architectures for syndromic and nonsyndromic congenital heart defects identified by exome sequencing
title_full Distinct genetic architectures for syndromic and nonsyndromic congenital heart defects identified by exome sequencing
title_fullStr Distinct genetic architectures for syndromic and nonsyndromic congenital heart defects identified by exome sequencing
title_full_unstemmed Distinct genetic architectures for syndromic and nonsyndromic congenital heart defects identified by exome sequencing
title_short Distinct genetic architectures for syndromic and nonsyndromic congenital heart defects identified by exome sequencing
title_sort distinct genetic architectures for syndromic and nonsyndromic congenital heart defects identified by exome sequencing
topic Clinical Genetics
Congenital Heart Defects
Genetics Research
url https://eprints.nottingham.ac.uk/38584/
https://eprints.nottingham.ac.uk/38584/
https://eprints.nottingham.ac.uk/38584/