Genetics in Medicine - 2018

258 articles | Last updated: 2025-12-03 14:12:56
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T A T A T A T A
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Laboratory analysis of amino acids, 2018 revision: a technical standard of the American College of Medical Genetics and Genomics (ACMG)
Venous thromboembolism laboratory testing (factor V Leiden andfactor II c.*97G>A), 2018 update: a technical standard of the American College of Medical Genetics and Genomics (ACMG)
Insuring patient access and affordability for treatments for rare and ultrarare diseases: a policy statement of the American College of Medical Genetics and Genomics
Medical genetics in developing countries in the Asia-Pacific region: challenges and opportunities
Discovery of new susceptibility genes: proceed cautiously
Correction: Population genomics in South East Asia captures unexpectedly high carrier frequency for treatable inherited disorders
The power of heredity and the relevance of eugenic history
Next-generation sequencing and prenatal 'omics: advanced diagnostics and new insights into human development
Role of MDH2 pathogenic variant in pheochromocytoma and paraganglioma patients
Landscape of pathogenic variations in a panel of 34 genes and cancer risk estimation from 5131 HBOC families
Navigating the nuances of clinical sequence variant interpretation in Mendelian disease
Utility and limitations of exome sequencing as a genetic diagnostic tool for children with hearing loss
Yield of additional genetic testing after chromosomal microarray for diagnosis of neurodevelopmental disability and congenital anomalies: a clinical practice resource of the American College of Medica
Genetic evaluation of cardiomyopathy: a clinical practice resource of the American College of Medical Genetics and Genomics (ACMG)
Genome-first findings require precision phenotyping
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News Briefs
Gain-of-function mutations in DNMT3A in patients with paraganglioma
Attention: Direct-To-Consumer patrons: Proceed with caution
Correction: In This Issue
Prenatal cell-free DNA screening test failures: a systematic review of failure rates, risks of Down syndrome, and impact of repeat testing
Phenotypic expansion illuminates multilocus pathogenic variation
Care of adults with neurofibromatosis type 1: a clinical practice resource of the American College of Medical Genetics and Genomics (ACMG)
Arno G. Motulsky, MD (1923–2018): Holocaust survivor who cofounded the field of medical genetics
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Analysis of 17 genes detects mutations in 81% of 811 patients with lissencephaly
Diversity of genetic events associated with MLH1 promoter methylation in Lynch syndrome families with heritable constitutional epimutation
Measuring coverage and accuracy of whole-exome sequencing in clinical context
Genomic sequencing identifies secondary findings in a cohort of parent study participants
Point-of-care whole-exome sequencing of idiopathic male infertility
Genetic disorders and mortality in infancy and early childhood: delayed diagnoses and missed opportunities
In This Issue
Correction: Corrigendum: Quantitative clinical characteristics of 53 patients with MPS VII: a cross-sectional analysis
News Briefs
Expanding the phenome and variome of skeletal dysplasia
In This Issue
AUDIOME: a tiered exome sequencing–based comprehensive gene panel for the diagnosis of heterogeneous nonsyndromic sensorineural hearing loss
FMR1 allele size distribution in 35,000 males and females: a comparison of developmental delay and general population cohorts
“Possibly positive or certainly uncertain?”: participants’ responses to uncertain diagnostic results from exome sequencing
Familial communication and cascade testing among relatives of BRCA population screening participants
Whole-exome sequencing reanalysis at 12 months boosts diagnosis and is cost-effective when applied early in Mendelian disorders
News Briefs
Genotype and phenotype correlation in von Hippel–Lindau disease based on alteration of the HIF-α binding site in VHL protein
Rapid prenatal diagnosis using targeted exome sequencing: a cohort study to assess feasibility and potential impact on prenatal counseling and pregnancy management
Exome sequencing in neonates: diagnostic rates, characteristics, and time to diagnosis
Short-term costs of integrating whole-genome sequencing into primary care and cardiology settings: a pilot randomized trial
Whole-genome sequencing offers additional but limited clinical utility compared with reanalysis of whole-exome sequencing
False-positive results released by direct-to-consumer genetic tests highlight the importance of clinical confirmation testing for appropriate patient care
Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective stu
Is prenatal genomic testing ready for prime time?
Risks of breast or ovarian cancer in BRCA1 or BRCA2 predictive test negatives: findings from the EMBRACE study
Patterns of homozygosity in patients with uniparental disomy: detection rate and suggested reporting thresholds for SNP microarrays
Pediatric clinical exome/genome sequencing and the engagement process: encouraging active conversation with the older child and adolescent: points to consider—a statement of the American College of Me
Laboratory analysis of organic acids, 2018 update: a technical standard of the American College of Medical Genetics and Genomics (ACMG)
Meeting the challenges of implementing rapid genomic testing in acute pediatric care
Response to Biesecker and Harrison
Congenital heart disease and aortic arch variants associated with mutation in PHOX2B
Targeted genetic analysis in a large cohort of familial and sporadic cases of aneurysm or dissection of the thoracic aorta
The ACMG/AMP reputable source criteria for the interpretation of sequence variants
Clinical and genetic heterogeneity in a large cohort of Armenian patients with late-onset familial Mediterranean fever
A point mutation in the pre-ZRS disrupts sonic hedgehog expression in the limb bud and results in triphalangeal thumb–polysyndactyly syndrome
Toward an effective exome-based genetic testing strategy in pediatric dilated cardiomyopathy
“Well, good luck with that”: reactions to learning of increased genetic risk for Alzheimer disease
Prenatal upper-limb mesomelia and 2q31.1 microdeletions affecting the regulatory genome
Mitochondrial oxodicarboxylate carrier deficiency is associated with mitochondrial DNA depletion and spinal muscular atrophy–like disease
Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison
Response to Peron et al.
Response to Walker
Tell me once, tell me soon: parents’ preferences for clinical genetics services for congenital heart disease
Anticipating uncertainty and irrevocable decisions: provider perspectives on implementing whole-genome sequencing in critically ill children with heart disease
Do RET somatic mutations play a role in Hirschsprung disease?
Hereditary cancer gene panel test reports: wide heterogeneity suggests need for standardization
ClinGen’s RASopathy Expert Panel consensus methods for variant interpretation
Mosaic maternal 10qter deletions are associated with FRA10B expansions and may cause false-positive noninvasive prenatal screening results
Cell-free fetal DNA versus maternal serum screening for trisomy 21 in pregnant women with and without assisted reproduction technology: a prospective interventional study
Response to Brosens et al
A counseling framework for moderate-penetrance colorectal cancer susceptibility genes
In This Issue
News Briefs
Autosomal recessive Noonan syndrome associated with biallelic LZTR1 variants
CLAPO syndrome: identification of somatic activating PIK3CA mutations and delineation of the natural history and phenotype
Are whole-exome and whole-genome sequencing approaches cost-effective? A systematic review of the literature
Correction: Novel findings with reassessment of exome data: implications for validation testing and interpretation of genomic data
SCYL1 variants cause a syndrome with lowγ-glutamyl-transferase cholestasis, acute liver failure, and neurodegeneration (CALFAN)
A metabolomic map of Zellweger spectrum disorders reveals novel disease biomarkers
Clinical whole-genome sequencing from routine formalin-fixed, paraffin-embedded specimens: pilot study for the 100,000 Genomes Project
Correction: Reproductive genetic carrier screening for cystic fibrosis, fragile X syndrome, and spinal muscular atrophy in Australia: outcomes of 12,000 tests
Making pretest genomic counseling optional: lessons from the RAVE study
Correction: Candidate-gene criteria for clinical reporting: diagnostic exome sequencing identifies altered candidate genes among 8% of patients with undiagnosed diseases
Interpretation of genomic sequencing: variants should be considered uncertain until proven guilty
Correction: Risk of colorectal cancer for carriers of a germ-line mutation in POLE or POLD1
A literature review at genome scale: improving clinical variant assessment
Clinical validity of phenotype-driven analysis software PhenoVar as a diagnostic aid for clinical geneticists in the interpretation of whole-exome sequencing data
Anticipated responses of early adopter genetic specialists and nongenetic specialists to unsolicited genomic secondary findings
Reconsidering the duty to warn genetically at-risk relatives
Considerations in healthcare reform for patients and families with genetic diseases: a statement of the American College of Medical Genetics and Genomics
CardioClassifier: disease- and gene-specific computational decision support for clinical genome interpretation
The contribution of family history to the burden of diagnosed diabetes, undiagnosed diabetes, and prediabetes in the United States: analysis of the National Health and Nutrition Examination Survey, 20
MSH6 and PMS2 germ-line pathogenic variants implicated in Lynch syndrome are associated with breast cancer
News Briefs
Erratum: In-depth investigations of adolescents and adults with holoprosencephaly identify unique characteristics
In This Issue
Making new genetic diagnoses with old data: iterative reanalysis and reporting from genome-wide data in 1,133 families with developmental disorders
Response to Wald et al
Arterial tortuosity syndrome: 40 new families and literature review
Professional responsibilities regarding the provision, publication, and dissemination of patient phenotypes in the context of clinical genetic and genomic testing: points to consider—a statement of th
Cost-effectiveness and comparative effectiveness of cancer risk management strategies in BRCA1/2 mutation carriers: a systematic review
Correction: Novel findings with reassessment of exome data: implications for validation testing and interpretation of genomic data
The differential diagnosis of a TP53 genetic testing result
Representing a “revolution”: how the popular press has portrayed personalized medicine
Phenotype and genotype of 87 patients with Mowat–Wilson syndrome and recommendations for care
Patient understanding of, satisfaction with, and perceived utility of whole-genome sequencing: findings from the MedSeq Project
A homozygous founder missense variant in arylsulfatase G abolishes its enzymatic activity causing atypical Usher syndrome in humans
Urine cell-free DNA is a biomarker for nephroblastomatosis or Wilms tumor in PIK3CA-related overgrowth spectrum (PROS)
Conflicts of interest in genetic counseling: addressing and delivering
Which Lynch syndrome screening programs could be implemented in the “real world”? A systematic review of economic evaluations
Clinical history and management recommendations of the smooth muscle dysfunction syndrome due to ACTA2 arginine 179 alterations
Adaptation and validation of the ACMG/AMP variant classification framework for MYH7-associated inherited cardiomyopathies: recommendations by ClinGen’s Inherited Cardiomyopathy Expert Panel
The role of SMAD3 in the genetic predisposition to papillary thyroid carcinoma
Proposed outcomes measures for state public health genomic programs
Conflicts of interest in genetic counseling: persistent underlying questions
Development and evaluation of a genomics training program for community health workers in Texas
Correction: Corrigendum: Autozygosity reveals recessive mutations and novel mechanisms in dominant genes: implications in variant interpretation
Modeling the ACMG/AMP variant classification guidelines as a Bayesian classification framework
The cost and diagnostic yield of exome sequencing for children with suspected genetic disorders: a benchmarking study
Cost-effectiveness analysis of germ-line BRCA testing in women with breast cancer and cascade testing in family members of mutation carriers
Genetic analysis of CHARGE syndrome identifies overlapping molecular biology
Correction: The genetic basis of classic nonketotic hyperglycinemia due to mutations in GLDC and AMT
CGG repeat length and AGG interruptions as indicators of fragile X–associated diminished ovarian reserve
Metaphors matter: from biobank to a library of medical information
Clinically impactful differences in variant interpretation between clinicians and testing laboratories: a single-center experience
A systematic analysis of online marketing materials used by providers of expanded carrier screening
From public health genomics to precision public health: a 20-year journey
Erratum: Laboratory diagnosis of biotinidase deficiency, 2017 update: a technical standard and guideline of the American College of Medical Genetics and Genomics
Response to Metcalfe et al.
Reinterpretation of sequence variants: one diagnostic laboratory’s experience, and the need for standard guidelines
Happy birthday, Genetics in Medicine!
Fragile X population carrier screening
Mosaic disorders and the Taxonomy of Human Disease
Prevalence of neurofibromatosis type 1 in the Finnish population
Correction: Corrigendum: Sources of discordance among germ-line variant classifications in ClinVar
Phenocopies in melanoma-prone families with germ-line CDKN2A mutations
Cherchez la femme: maternal incidental findings can explain discordant prenatal cell-free DNA sequencing results
Follow-up status during the first 5 years of life for metabolic disorders on the federal Recommended Uniform Screening Panel
Age and perceived risks and benefits of preventive genomic screening
Observed frequency and challenges of variant reclassification in a hereditary cancer clinic
A craniosynostosis massively parallel sequencing panel study in 309 Australian and New Zealand patients: findings and recommendations
Coronary calcification in adults with Turner syndrome
Somatic TP53 variants frequently confound germ-line testing results
Origin and clinical relevance of chromosomal aberrations other than the common trisomies detected by genome-wide NIPS: results of the TRIDENT study
Ashkenazi Jewish genomic variants: integrating data from the Israeli National Genetic Database and gnomAD
Evaluating CHARGE syndrome in congenital hypogonadotropic hypogonadism patients harboring CHD7 variants
Characterizing reduced coverage regions through comparison of exome and genome sequencing data across 10 centers
De novo variants in the alternative exon 5 of SCN8A cause epileptic encephalopathy
Prenatal reflex DNA screening for trisomies 21, 18, and 13
Risk of colorectal cancer for carriers of a germ-line mutation in POLE or POLD1
Informative priors on fetal fraction increase power of the noninvasive prenatal screen
Precision newborn screening for lysosomal disorders
Peter Bauer, Ellen Karges, Gabriela Oprea and Arndt Rolfs
Moonlighting newborn screening markers: the incidental discovery of a second-tier test for Pompe disease
Proband-only medical exome sequencing as a cost-effective first-tier genetic diagnostic test for patients without prior molecular tests and clinical diagnosis in a developing country: the China experi
Clinical whole-exome sequencing for the diagnosis of rare disorders with congenital anomalies and/or intellectual disability: substantial interest of prospective annual reanalysis
Identification of balanced chromosomal rearrangements previously unknown among participants in the 1000 Genomes Project: implications for interpretation of structural variation in genomes and the futu
School liaison program supporting children with neurofibromatosis type 1: a model of care for children with chronic disease
Correction: Corrigendum: What are people willing to pay for whole-genome sequencing information, and who decides what they receive?
Unmet needs in human genomic variant interpretation
Revisiting mitochondrial diagnostic criteria in the new era of genomics
Reproductive genetic carrier screening for cystic fibrosis, fragile X syndrome, and spinal muscular atrophy in Australia: outcomes of 12,000 tests
Novel copy-number variations in pharmacogenes contribute to interindividual differences in drug pharmacokinetics
Biallelic and monoallelic ESR2 variants associated with 46,XY disorders of sex development
Laboratory diagnosis of galactosemia: a technical standard and guideline of the American College of Medical Genetics and Genomics (ACMG)
Genomic study of severe fetal anomalies and discovery of GREB1L mutations in renal agenesis
The who, what, and why of research participants’ intentions to request a broad range of secondary findings in a diagnostic genomic sequencing study
RET somatic mutations are underrecognized in Hirschsprung disease
Early cancer diagnoses through BRCA1/2 screening of unselected adult biobank participants
Identification of SLC20A1 and SLC15A4 among other genes as potential risk factors for combined pituitary hormone deficiency
Beware the laboratory report: discrepancy in variant classification on reproductive carrier screening
Analysis of publicly available LDLR, APOB, and PCSK9 variants associated with familial hypercholesterolemia: application of ACMG guidelines and implications for familial hypercholesterolemia diagnosis
“Not pathogenic until proven otherwise”: perspectives of UK clinical genomics professionals toward secondary findings in context of a Genomic Medicine Multidisciplinary Team and the 100,000 Genomes Pr
Catching hidden variation: systematic correction of reference minor allele annotation in clinical variant calling
Low frequency of Fabry disease in patients with common heart disease
Characterization of pulmonary arteriovenous malformations in ACVRL1 versus ENG mutation carriers in hereditary hemorrhagic telangiectasia
Recommending inclusion of HFE C282Y homozygotes in the ACMG actionable gene list: cop-out or stealth move toward population screening?
A cross-sectional quantitative analysis of the natural history of Farber disease: an ultra-orphan condition with rheumatologic and neurological cardinal disease features
Recurrent variants in OTOF are significant contributors to prelingual nonsydromic hearing loss in Saudi patients
Pathways from autism spectrum disorder diagnosis to genetic testing
Pilot study of population-based newborn screening for spinal muscular atrophy in New York state
A randomized trial to study the comparative efficacy of phenylbutyrate and benzoate on nitrogen excretion and ureagenesis in healthy volunteers
A C-terminal nonsense mutation links PTPRQ with autosomal-dominant hearing loss, DFNA73
Clinical relevance of systematic phenotyping and exome sequencing in patients with short stature
Noncoding copy-number variations are associated with congenital limb malformation
Monogenic diabetes in overweight and obese youth diagnosed with type 2 diabetes: the TODAY clinical trial
Novel findings with reassessment of exome data: implications for validation testing and interpretation of genomic data
Vascular phenotypes in nonvascular subtypes of the Ehlers-Danlos syndrome: a systematic review
A common haplotype containing functional CACNA1H variants is frequently coinherited with increased TPSAB1 copy number
Trends in utilization and costs of BRCA testing among women aged 18–64 years in the United States, 2003–2014
Developing a common framework for evaluating the implementation of genomic medicine interventions in clinical care: the IGNITE Network’s Common Measures Working Group
Comprehensive use of extended exome analysis improves diagnostic yield in rare disease: a retrospective survey in 1,059 cases
Looking beyond the exome: a phenotype-first approach to molecular diagnostic resolution in rare and undiagnosed diseases
Impact of HIPAA’s minimum necessary standard on genomic data sharing
Clinical and genetic analysis of a rare syndrome associated with neoteny
Navigating the research–clinical interface in genomic medicine: analysis from the CSER Consortium
Individuals with FANCM biallelic mutations do not develop Fanconi anemia, but show risk for breast cancer, chemotherapy toxicity and may display chromosome fragility
High frequency of mosaic pathogenic variants in genes causing epilepsy-related neurodevelopmental disorders
Biallelic truncating FANCM mutations cause early-onset cancer but not Fanconi anemia
Biallelic variants in LINGO1 are associated with autosomal recessive intellectual disability, microcephaly, speech and motor delay
Inferred inheritance of MorbidMap genes without OMIM clinical synopsis
Monoallelic and biallelic CREB3L1 variant causes mild and severe osteogenesis imperfecta, respectively
Prospective cohort study for identification of underlying genetic causes in neonatal encephalopathy using whole-exome sequencing
Genetic disruption of the oncogenic HMGA2–PLAG1–IGF2 pathway causes fetal growth restriction
A proposed approach to accelerate evidence generation for genomic-based technologies in the context of a learning health system
B3GAT3-related disorder with craniosynostosis and bone fragility due to a unique mutation
Clinical penetrance in hereditary hemochromatosis: estimates of the cumulative incidence of severe liver disease among HFE C282Y homozygotes
De novo and rare inherited copy-number variations in the hemiplegic form of cerebral palsy
Improved diagnostic yield compared with targeted gene sequencing panels suggests a role for whole-genome sequencing as a first-tier genetic test
Precision medicine, health disparities, and ethics: the case for disability inclusion
Multiplex epithelium dysfunction due to CLDN10 mutation: the HELIX syndrome
Parental preferences toward genomic sequencing for non-medically actionable conditions in children: a discrete-choice experiment
Interpreting the clinical significance of combined variants in multiple recessive disease genes: systematic investigation of Joubert syndrome yields little support for oligogenicity
Reactions to clinical reinterpretation of a gene variant by participants in a sequencing study
SLC39A8 deficiency: biochemical correction and major clinical improvement by manganese therapy
Racial/ethnic differences in multiple-gene sequencing results for hereditary cancer risk
Mapping the genomic landscape of inherited retinal disease genes prioritizes genes prone to coding and noncoding copy-number variations
The wide genetic landscape of clinical frontotemporal dementia: systematic combined sequencing of 121 consecutive subjects
Molecular autopsy in maternal–fetal medicine
A patient with germ-line gain-of-function PDGFRB p.N666H mutation and marked clinical response to imatinib
When genotype is not predictive of phenotype: implications for genetic counseling based on 21,594 chromosomal microarray analysis examinations
A randomized controlled trial of disclosing genetic risk information for Alzheimer disease via telephone
Genomic diagnostics within a medically underserved population: efficacy and implications
Quality of life in men with Klinefelter syndrome: the impact of genotype, health, socioeconomics, and sexual function
Knowledge base and mini-expert platform for the diagnosis of inborn errors of metabolism
PREPL deficiency: delineation of the phenotype and development of a functional blood assay
Prevalence of germ-line mutations in cancer genes among pancreatic cancer patients with a positive family history
Data sharing as a national quality improvement program: reporting on BRCA1 and BRCA2 variant-interpretation comparisons through the Canadian Open Genetics Repository (COGR)
Survival trends from the Prader–Willi Syndrome Association (USA) 40-year mortality survey
CAP/ACMG proficiency testing for biochemical genetics laboratories: a summary of performance
Mutations in C-natriuretic peptide (NPPC): a novel cause of autosomal dominant short stature
FOXG1 syndrome: genotype–phenotype association in 83 patients with FOXG1 variants
GWAS signals revisited using human knockouts
In-depth investigations of adolescents and adults with holoprosencephaly identify unique characteristics
Long-read genome sequencing identifies causal structural variation in a Mendelian disease
Broad consent for health care–embedded biobanking: understanding and reasons to donate in a large patient sample
Systematic design and comparison of expanded carrier screening panels
Genetic testing impacts the utility of prospective familial screening in hypertrophic cardiomyopathy through identification of a nonfamilial subgroup
A cohort of 17 patients with kyphoscoliotic Ehlers–Danlos syndrome caused by biallelic mutations in FKBP14: expansion of the clinical and mutational spectrum and description of the natural history