ClinGen--the Clinical Genome Resource.

On autopsy, a patient is found to have hypertrophic cardiomyopathy. The patient’s family pursues genetic testing that shows a “likely pathogenic” variant for the condition on the basis of a study in an original research publication. Given the dominant inheritance of the condition and the risk of sudden cardiac death, other family members are tested for the genetic variant to determine their risk. Several family members test negative and are told that they are not at risk for hypertrophic cardiomyopathy and sudden cardiac death, and those who test positive are told that they need to be regularly monitored for cardiomyopathy on echocardiography. Five years later, during a routine clinic visit of one of the genotype-positive family members, the cardiologist queries a database for current knowledge on the genetic variant and discovers that the variant is now interpreted as “likely benign” by another laboratory that uses more recently derived population-frequency data. A newly available testing panel for additional genes that are implicated in hypertrophic cardiomyopathy is initiated on an affected family member, and a different variant is found that is determined to be pathogenic. Family members are retested, and one member who previously tested negative is now found to be positive for this new variant. An immediate clinical workup detects evidence of cardiomyopathy, and an intracardiac defibrillator is implanted to reduce the risk of sudden cardiac death.

[1]  D. Valle,et al.  PhenoDB: A New Web-Based Tool for the Collection, Storage, and Analysis of Phenotypic Features , 2013, Human mutation.

[2]  Leslie G Biesecker,et al.  Consensus statement: chromosomal microarray is a first-tier clinical diagnostic test for individuals with developmental disabilities or congenital anomalies. , 2010, American journal of human genetics.

[3]  James P Evans,et al.  An informatics approach to analyzing the incidentalome , 2012, Genetics in Medicine.

[4]  Gail P Jarvik,et al.  The FDA and genomic tests--getting regulation right. , 2015, The New England journal of medicine.

[5]  J. Svendsen,et al.  New population-based exome data are questioning the pathogenicity of previously cardiomyopathy-associated genetic variants , 2013, European Journal of Human Genetics.

[6]  George P Patrinos,et al.  Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene , 2013, Nature Genetics.

[7]  Nazneen Rahman,et al.  Gene-panel sequencing and the prediction of breast-cancer risk. , 2015, The New England journal of medicine.

[8]  Russ B Altman,et al.  PharmGKB: the pharmacogenetics and pharmacogenomics knowledge base. , 2005, Methods in molecular biology.

[9]  Russ B Altman,et al.  PharmGKB: the Pharmacogenomics Knowledge Base. , 2013, Methods in molecular biology.

[10]  Ncbi National Center for Biotechnology Information , 2008 .

[11]  Teri A. Manolio,et al.  Bringing genome-wide association findings into clinical use , 2013, Nature Reviews Genetics.

[12]  Rodney J Scott,et al.  Application of a 5-tiered scheme for standardized classification of 2,360 unique mismatch repair gene variants in the InSiGHT locus-specific database , 2013, Nature Genetics.

[13]  Hilary S. Leeds,et al.  Data use under the NIH GWAS Data Sharing Policy and future directions , 2014, Nature Genetics.

[14]  Michael Brudno,et al.  PhenoTips: Patient Phenotyping Software for Clinical and Research Use , 2013, Human mutation.

[15]  Heidi L. Rehm,et al.  Communicating new knowledge on previously reported genetic variants , 2012, Genetics in Medicine.

[16]  Deanna M. Church,et al.  ClinVar: public archive of relationships among sequence variation and human phenotype , 2013, Nucleic Acids Res..

[17]  P. Donnelly,et al.  Replicating genotype–phenotype associations , 2007, Nature.

[18]  Ryan W. Kim,et al.  Carrier Testing for Severe Childhood Recessive Diseases by Next-Generation Sequencing , 2011, Science Translational Medicine.

[19]  Bale,et al.  Standards and Guidelines for the Interpretation of Sequence Variants: A Joint Consensus Recommendation of the American College of Medical Genetics and Genomics and the Association for Molecular Pathology , 2015, Genetics in Medicine.

[20]  Barry J Maron,et al.  2011 ACCF/AHA guideline for the diagnosis and treatment of hypertrophic cardiomyopathy: executive summary: a report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines. , 2011, Journal of the American College of Cardiology.

[21]  S. South,et al.  American College of Medical Genetics standards and guidelines for interpretation and reporting of postnatal constitutional copy number variants , 2011, Genetics in Medicine.

[22]  Sue Povey,et al.  Sharing data between LSDBs and central repositories , 2009, Human mutation.

[23]  Barry J Maron,et al.  2011 ACCF/AHA Guideline for the Diagnosis and Treatment of Hypertrophic Cardiomyopathy: a report of the American College of Cardiology Foundation/American Heart Association Task Force on Practice Guidelines. Developed in collaboration with the American Association for Thoracic Surgery, American Soci , 2011, Journal of the American College of Cardiology.

[24]  D. Longo,et al.  Precision medicine--personalized, problematic, and promising. , 2015, The New England journal of medicine.

[25]  S. Eddy,et al.  Sharing Publication-Related Data and Materials: Responsibilities of Authorship in the Life Sciences1 , 2003, Plant Physiology.