Recommendations by the ClinGen Rett/Angelman-like expert panel for gene-specific variant interpretation methods.

Angelman syndrome Christianson syndrome Pitt-Hopkins syndrome Rett syndrome guidelines variant interpretation

Journal

Human mutation
ISSN: 1098-1004
Titre abrégé: Hum Mutat
Pays: United States
ID NLM: 9215429

Informations de publication

Date de publication:
08 2022
Historique:
revised: 05 11 2021
received: 19 07 2021
accepted: 21 11 2021
pubmed: 28 11 2021
medline: 20 7 2022
entrez: 27 11 2021
Statut: ppublish

Résumé

The genes MECP2, CDKL5, FOXG1, UBE3A, SLC9A6, and TCF4 present unique challenges for current ACMG/AMP variant interpretation guidelines. To address those challenges, the Rett and Angelman-like Disorders Variant Curation Expert Panel (Rett/AS VCEP) drafted gene-specific modifications. A pilot study was conducted to test the clarity and accuracy of using the customized variant interpretation criteria. Multiple curators obtained the same interpretation for 78 out of the 87 variants (~90%), indicating appropriate usage of the modified guidelines the majority of times by all the curators. The classification of 13 variants changed using these criteria specifications compared to when the variants were originally curated and as present in ClinVar. Many of these changes were due to internal data shared from laboratory members however some changes were because of changes in strength of criteria. There were no two-step classification changes and only 1 clinically relevant change (Likely pathogenic to VUS). The Rett/AS VCEP hopes that these gene-specific variant curation rules and the assertions provided help clinicians, clinical laboratories, and others interpret variants in these genes but also other fully penetrant, early-onset genes associated with rare disorders.

Identifiants

pubmed: 34837432
doi: 10.1002/humu.24302
pmc: PMC9135956
mid: NIHMS1759624
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't Research Support, N.I.H., Extramural

Langues

eng

Sous-ensembles de citation

IM

Pagination

1097-1113

Subventions

Organisme : NICHD NIH HHS
ID : P50 HD103537
Pays : United States
Organisme : NHGRI NIH HHS
ID : U24 HG006834
Pays : United States
Organisme : NHGRI NIH HHS
ID : U41 HG006834
Pays : United States

Informations de copyright

© 2021 Wiley Periodicals LLC.

Références

Am J Med Genet A. 2004 Apr 15;126A(2):129-40
pubmed: 15057977
BMC Med Genet. 2014 Feb 25;15:24
pubmed: 24564546
Neuron. 1998 Oct;21(4):799-811
pubmed: 9808466
Genet Med. 2015 May;17(5):405-24
pubmed: 25741868
BMC Med Genet. 2012 Aug 06;13:68
pubmed: 22867051
Eur J Hum Genet. 2014 Feb;22(2):270-2
pubmed: 23756444
Ann Neurol. 2020 Aug;88(2):396-406
pubmed: 32472944
Nat Neurosci. 2013 Jul;16(7):898-902
pubmed: 23770565
Hum Mutat. 2012 Aug;33(8):1228-38
pubmed: 22505045
Hum Mutat. 2017 Aug;38(8):922-931
pubmed: 28544139
Eur J Med Genet. 2012 Dec;55(12):677-81
pubmed: 22982301
Ann Neurol. 2010 Dec;68(6):944-50
pubmed: 21154482
Am J Cardiol. 1989 Jan 15;63(3):249-50
pubmed: 2521274
Clin Genet. 2019 Apr;95(4):462-478
pubmed: 30677142
Am J Hum Genet. 2008 Apr;82(4):1003-10
pubmed: 18342287
Neurol Genet. 2018 Nov 07;4(6):e281
pubmed: 30533527
Clin Genet. 2016 Jun;89(6):733-8
pubmed: 26936630
Epilepsia. 2019 Jan;60(1):155-164
pubmed: 30525188
Genet Med. 2018 Sep;20(9):1054-1060
pubmed: 29300386
Hum Mutat. 2014 Dec;35(12):1407-17
pubmed: 25212744
Genet Med. 2018 Dec;20(12):1687-1688
pubmed: 29543229
Am J Hum Genet. 2008 Jul;83(1):89-93
pubmed: 18571142
Brain Dev. 2005 Apr;27(3):211-7
pubmed: 15737703
Clin Genet. 2009 Oct;76(4):357-71
pubmed: 19793311
Nat Genet. 2001 Mar;27(3):322-6
pubmed: 11242117
Hum Mutat. 2018 Nov;39(11):1581-1592
pubmed: 30311380
Hum Mutat. 2018 Nov;39(11):1517-1524
pubmed: 30192042
Genet Med. 2018 Mar;20(3):351-359
pubmed: 29300372
Proc Natl Acad Sci U S A. 2012 Dec 26;109(52):21516-21
pubmed: 23236174
Eur J Med Genet. 2006 Jan-Feb;49(1):9-18
pubmed: 16473305
Hum Genet. 2012 Feb;131(2):187-200
pubmed: 21748340
Hum Mutat. 2018 Nov;39(11):1553-1568
pubmed: 30311375
Mol Cell Biol. 1996 Jun;16(6):2898-905
pubmed: 8649400
Neurol Genet. 2017 Dec 15;3(6):e200
pubmed: 29264392
Nat Genet. 2009 May;41(5):535-43
pubmed: 19377476
Eur Child Adolesc Psychiatry. 2001 Jun;10(2):117-21
pubmed: 11469283
J Med Genet. 2008 Nov;45(11):738-44
pubmed: 18728071
Genet Med. 2018 Nov;20(11):1334-1345
pubmed: 29493581
Genome Med. 2019 Dec 31;12(1):3
pubmed: 31892348
Hum Mol Genet. 2000 May 22;9(9):1377-84
pubmed: 10814719
Sci Rep. 2016 Dec 08;6:38590
pubmed: 27929079
Hum Mutat. 2012 Jan;33(1):64-72
pubmed: 22045651
Am J Med Genet A. 2018 Jul;176(7):1641-1647
pubmed: 29737008
Brain Sci. 2020 Feb 17;10(2):
pubmed: 32079229
Hum Mutat. 2018 Nov;39(11):1593-1613
pubmed: 30311386
Am J Hum Genet. 2016 Oct 6;99(4):877-885
pubmed: 27666373
Science. 2004 Jan 2;303(5654):56-9
pubmed: 14704420
Brain. 2011 Nov;134(Pt 11):3369-83
pubmed: 21964919
Eur J Med Genet. 2016 Oct;59(10):522-5
pubmed: 27465203
Gene. 2013 Jan 1;512(1):70-5
pubmed: 23064044
Blood Adv. 2019 Oct 22;3(20):2962-2979
pubmed: 31648317
Genet Med. 2018 Jan;20(1):98-108
pubmed: 28661489
Eur J Hum Genet. 2018 Jul;26(7):996-1006
pubmed: 29695756
J Med Genet. 2008 Mar;45(3):172-8
pubmed: 17993579
Biochem Cell Biol. 2011 Feb;89(1):1-11
pubmed: 21326358
Hum Genet. 2016 Aug;135(8):841-50
pubmed: 27142213
Hum Mol Genet. 1999 Jan;8(1):129-35
pubmed: 9887341
J Med Genet. 2017 Feb;54(2):87-92
pubmed: 27620904
Hum Genet. 2010 Jan;127(1):118
pubmed: 20108430
Clin Genet. 2002 May;61(5):359-62
pubmed: 12081720
Am J Med Genet A. 2016 Aug;170(8):2103-10
pubmed: 27256868
Eur J Hum Genet. 2013 Mar;21(3):266-73
pubmed: 22872100
Am J Hum Genet. 2007 May;80(5):988-93
pubmed: 17436254
Hum Genome Var. 2019 Mar 25;6:15
pubmed: 30937176
Proc Natl Acad Sci U S A. 2007 Sep 25;104(39):15382-7
pubmed: 17878293
Epilepsia. 2018 May;59(5):1062-1071
pubmed: 29655203

Auteurs

Dianalee McKnight (D)

Invitae, San Francisco, California, USA.

Lora Bean (L)

Perkin Elmer, Duluth, Georgia, USA.

Izabela Karbassi (I)

Athena Diagnostics, Worcester, Massachusetts, USA.

Katelynn Beattie (K)

GeneDx, Gaithersburg, Massachusetts, USA.

Thierry Bienvenu (T)

Institut de Psychiatrie et de Neurosciences de Paris (IPNP), Paris, France.

Hope Bonin (H)

Central Manchester University Hospital, Manchester, Greater Manchester, UK.

Ping Fang (P)

Baylor College of Medicine, Houston, Texas, USA.

John Chrisodoulou (J)

Murdoch Childrens Research Institute and the University of Melbourne, Parkville, Victoria, Australia.
University of Sydney, Sydney, New South Wales, Australia.

Michael Friez (M)

Greenwood Genetic Center, Greenwood, South Carolina, USA.

Maria Helgeson (M)

Invitae, San Francisco, California, USA.

Rahul Krishnaraj (R)

University of Sydney, Sydney, New South Wales, Australia.
Children's Hospital at Westmead, Westmead, New South Wales, Australia.

Linyan Meng (L)

Baylor College of Medicine, Houston, Texas, USA.

Lindsey Mighion (L)

GeneDx, Gaithersburg, Massachusetts, USA.

Jeffrey Neul (J)

Vanderbilt Kennedy Center, Nashville, Tennessee, USA.

Alan Percy (A)

University of Alabama at Birmingham, Birmingham, Alabama, USA.

Simon Ramsden (S)

Central Manchester University Hospital, Manchester, Greater Manchester, UK.

Huda Zoghbi (H)

Baylor College of Medicine, Houston, Texas, USA.

Soma Das (S)

University of Chicago, Chicago, Illinois, USA.

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