Updated ACMG/AMP specifications for variant interpretation and gene curations from the ClinGen RASopathy expert panels

Purpose: The ClinGen RASopathy (RAS) Variant Curation Expert Panel (VCEP) previously established RASopathy specifications to the American College of Medical Genetics and Genomics (ACMG) and Association of Molecular Pathology (AMP) variant classification framework for more consistent and accurate var...

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Main Authors: Emma H. Wilcox, Ryan F. Webb, Kezang C. Tshering, Madeline Y. Hughes, Hélène Cavé, Marina T. DiStefano, Hannah Dziadzio, Kate Garber, Bruce D. Gelb, Karen W. Gripp, Shoji Ichikawa, Jennifer A. Lee, Hannah McCurry, Marco Tartaglia, Bradley Williams, Martin Zenker, Lisa M. Vincent, Heather Mason-Suares, Bruce Gelb, Jennifer Lee, Karen Gripp, Kat Lafferty, Kezang Tshering, Lisa Vincent, Luis Enrique Gomez, Marina DiStefano, Reza Ahmadian, Ryan Webb
Format: Article
Language:English
Published: Elsevier 2025-01-01
Series:Genetics in Medicine Open
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Online Access:http://www.sciencedirect.com/science/article/pii/S2949774425014694
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author Emma H. Wilcox
Ryan F. Webb
Kezang C. Tshering
Madeline Y. Hughes
Hélène Cavé
Marina T. DiStefano
Hannah Dziadzio
Kate Garber
Bruce D. Gelb
Karen W. Gripp
Shoji Ichikawa
Jennifer A. Lee
Hannah McCurry
Marco Tartaglia
Bradley Williams
Martin Zenker
Lisa M. Vincent
Heather Mason-Suares
Bradley Williams
Bruce Gelb
Hannah Dziadzio
Heather Mason-Suares
Hélène Cavé
Jennifer Lee
Karen Gripp
Kat Lafferty
Kezang Tshering
Lisa Vincent
Luis Enrique Gomez
Marco Tartaglia
Marina DiStefano
Martin Zenker
Reza Ahmadian
Ryan Webb
Shoji Ichikawa
author_facet Emma H. Wilcox
Ryan F. Webb
Kezang C. Tshering
Madeline Y. Hughes
Hélène Cavé
Marina T. DiStefano
Hannah Dziadzio
Kate Garber
Bruce D. Gelb
Karen W. Gripp
Shoji Ichikawa
Jennifer A. Lee
Hannah McCurry
Marco Tartaglia
Bradley Williams
Martin Zenker
Lisa M. Vincent
Heather Mason-Suares
Bradley Williams
Bruce Gelb
Hannah Dziadzio
Heather Mason-Suares
Hélène Cavé
Jennifer Lee
Karen Gripp
Kat Lafferty
Kezang Tshering
Lisa Vincent
Luis Enrique Gomez
Marco Tartaglia
Marina DiStefano
Martin Zenker
Reza Ahmadian
Ryan Webb
Shoji Ichikawa
author_sort Emma H. Wilcox
collection DOAJ
description Purpose: The ClinGen RASopathy (RAS) Variant Curation Expert Panel (VCEP) previously established RASopathy specifications to the American College of Medical Genetics and Genomics (ACMG) and Association of Molecular Pathology (AMP) variant classification framework for more consistent and accurate variant classification. Advances in the understanding of RASopathies and new clinical genetic testing algorithms required updated specifications. Methods: The RAS Gene Curation Expert Panel recurated 6 gene-disease relationships, and the RAS VCEP evaluated the previous specifications to develop updated RASopathy specifications for the ACMG/AMP framework. The performance of these updated specifications was tested by reassessing 59 previously classified variants and 88 new pilot variants. Results: Five gene-disease relationships were upgraded to Definitive, whereas 1 was upgraded to Moderate. Updated specifications were applied to 11 ACMG/AMP criteria for disorders with a dominant inheritance, 3 criteria for recessive inheritance, and 4 criteria to align with recommendations from the ClinGen Sequence Variant Interpretation Working Group. Assessment of variants demonstrated no major shifts in classifications compared with previous RAS VCEP or ClinVar classifications. Conclusion: Updated RASopathy specifications improve the classification of variants associated with recessive disease and observed in exome/genome cases. Most of these specifications may also be used as a baseline for other rare Mendelian disorders.
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spelling doaj-art-b78eaa9881a3495fa8c8d5da9e6a499c2025-08-20T02:26:10ZengElsevierGenetics in Medicine Open2949-77442025-01-01310343010.1016/j.gimo.2025.103430Updated ACMG/AMP specifications for variant interpretation and gene curations from the ClinGen RASopathy expert panelsEmma H. Wilcox0Ryan F. Webb1Kezang C. Tshering2Madeline Y. Hughes3Hélène Cavé4Marina T. DiStefano5Hannah Dziadzio6Kate Garber7Bruce D. Gelb8Karen W. Gripp9Shoji Ichikawa10Jennifer A. Lee11Hannah McCurry12Marco Tartaglia13Bradley Williams14Martin Zenker15Lisa M. Vincent16Heather Mason-Suares17Bradley WilliamsBruce GelbHannah DziadzioHeather Mason-SuaresHélène CavéJennifer LeeKaren GrippKat LaffertyKezang TsheringLisa VincentLuis Enrique GomezMarco TartagliaMarina DiStefanoMartin ZenkerReza AhmadianRyan WebbShoji IchikawaMedical and Population Genetics Program, Broad Institute of MIT and Harvard, Cambridge, MA; The Warren Alpert Medical School of Brown University, Providence, RIMedical and Population Genetics Program, Broad Institute of MIT and Harvard, Cambridge, MAMedical and Population Genetics Program, Broad Institute of MIT and Harvard, Cambridge, MAMedical and Population Genetics Program, Broad Institute of MIT and Harvard, Cambridge, MADépartement de Génétique, Hôpital Robert Debré (AP-HP), and Université Paris-Cité, Paris, FranceMedical and Population Genetics Program, Broad Institute of MIT and Harvard, Cambridge, MAMedical and Population Genetics Program, Broad Institute of MIT and Harvard, Cambridge, MADepartment of Human Genetics, Emory School of Medicine, Atlanta, GAMindich Child Health and Development Institute, Icahn School of Medicine at Mount Sinai, New York, NYDivision of Medical Genetics, Nemours Children’s Hospital, Wilmington, DEAmbry Genetics, Aliso Viejo, CAGreenwood Genetic Center, Greenwood, SCMedical and Population Genetics Program, Broad Institute of MIT and Harvard, Cambridge, MAOspedale Pediatrico Bambino Gesù, IRCCS, Rome, ItalyGeneDx, Gaithersburg, MDInstitute of Human Genetics, University Hospital Magdeburg, Magdeburg, GermanyNatera Inc, Austin, TXMass General Brigham, Laboratory for Molecular Medicine, Cambridge, MA; Correspondence and requests for materials should be addressed to Heather Mason-Suares, Laboratory for Molecular Medicine, 65 Landsdowne Street, Cambridge, MA 02139.Purpose: The ClinGen RASopathy (RAS) Variant Curation Expert Panel (VCEP) previously established RASopathy specifications to the American College of Medical Genetics and Genomics (ACMG) and Association of Molecular Pathology (AMP) variant classification framework for more consistent and accurate variant classification. Advances in the understanding of RASopathies and new clinical genetic testing algorithms required updated specifications. Methods: The RAS Gene Curation Expert Panel recurated 6 gene-disease relationships, and the RAS VCEP evaluated the previous specifications to develop updated RASopathy specifications for the ACMG/AMP framework. The performance of these updated specifications was tested by reassessing 59 previously classified variants and 88 new pilot variants. Results: Five gene-disease relationships were upgraded to Definitive, whereas 1 was upgraded to Moderate. Updated specifications were applied to 11 ACMG/AMP criteria for disorders with a dominant inheritance, 3 criteria for recessive inheritance, and 4 criteria to align with recommendations from the ClinGen Sequence Variant Interpretation Working Group. Assessment of variants demonstrated no major shifts in classifications compared with previous RAS VCEP or ClinVar classifications. Conclusion: Updated RASopathy specifications improve the classification of variants associated with recessive disease and observed in exome/genome cases. Most of these specifications may also be used as a baseline for other rare Mendelian disorders.http://www.sciencedirect.com/science/article/pii/S2949774425014694ClinGenNoonanRas/MAPKRASopathyVariant interpretation
spellingShingle Emma H. Wilcox
Ryan F. Webb
Kezang C. Tshering
Madeline Y. Hughes
Hélène Cavé
Marina T. DiStefano
Hannah Dziadzio
Kate Garber
Bruce D. Gelb
Karen W. Gripp
Shoji Ichikawa
Jennifer A. Lee
Hannah McCurry
Marco Tartaglia
Bradley Williams
Martin Zenker
Lisa M. Vincent
Heather Mason-Suares
Bradley Williams
Bruce Gelb
Hannah Dziadzio
Heather Mason-Suares
Hélène Cavé
Jennifer Lee
Karen Gripp
Kat Lafferty
Kezang Tshering
Lisa Vincent
Luis Enrique Gomez
Marco Tartaglia
Marina DiStefano
Martin Zenker
Reza Ahmadian
Ryan Webb
Shoji Ichikawa
Updated ACMG/AMP specifications for variant interpretation and gene curations from the ClinGen RASopathy expert panels
Genetics in Medicine Open
ClinGen
Noonan
Ras/MAPK
RASopathy
Variant interpretation
title Updated ACMG/AMP specifications for variant interpretation and gene curations from the ClinGen RASopathy expert panels
title_full Updated ACMG/AMP specifications for variant interpretation and gene curations from the ClinGen RASopathy expert panels
title_fullStr Updated ACMG/AMP specifications for variant interpretation and gene curations from the ClinGen RASopathy expert panels
title_full_unstemmed Updated ACMG/AMP specifications for variant interpretation and gene curations from the ClinGen RASopathy expert panels
title_short Updated ACMG/AMP specifications for variant interpretation and gene curations from the ClinGen RASopathy expert panels
title_sort updated acmg amp specifications for variant interpretation and gene curations from the clingen rasopathy expert panels
topic ClinGen
Noonan
Ras/MAPK
RASopathy
Variant interpretation
url http://www.sciencedirect.com/science/article/pii/S2949774425014694
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