Journal of Medical Genetics - 2019

117 articles | Last updated: 2025-12-03 14:12:57
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T A T A T A T A
Homozygosity for CHEK2 p.Gly167Arg leads to a unique cancer syndrome with multiple complex chromosomal translocations in peripheral blood karyotype
Increased<i>TBX6</i>gene dosages induce congenital cervical vertebral malformations in humans and mice
Clinical utility of NGS diagnosis and disease stratification in a multiethnic primary ciliary dyskinesia cohort white European
Multiregion whole-genome sequencing depicts intratumour heterogeneity and punctuated evolution in ovarian clear cell carcinoma
Mutations in gene regulatory elements linked to human limb malformations
A novel autosomal recessive lipodystrophy syndrome due to homozygous<i>LMNA</i>variant Hispanic
Gene editing prospects for treating inherited retinal diseases
Linked-read genome sequencing identifies biallelic pathogenic variants in <i>DONSON</i> as a novel cause of Meier-Gorlin syndrome
Retesting of women who are negative for a<i>BRCA1</i>and<i>BRCA2</i>mutation using a 20-gene panel
Three-step site-directed mutagenesis screen identifies pathogenic <i>MLH1</i> variants associated with Lynch syndrome
Genetic variability and potential effects on clinical trial outcomes: perspectives in Parkinson’s disease 5851 patients with Parkinson’s disease of European ancestry data
Deep phenotyping, including quantitative ciliary beating parameters, and extensive genotyping in primary ciliary dyskinesia
Cardiac valve involvement in <i>ADAR</i>-related type I interferonopathy
Review of: ‘Bone dysplasia—an atlas of genetic disorders of skeletal development (fourth edition)’ Edited by: Jürgen W Spranger, Paula W Brill, Christine Hall, Gen Nishimura, Andrea Superti-Furga, She
Biallelic VPS35L pathogenic variants cause 3C/Ritscher-Schinzel-like syndrome through dysfunction of retriever complex
Psychosocial effects of whole-body MRI screening in adult high-risk pathogenic<i>TP53</i>mutation carriers: a case-controlled study (SIGNIFY)
Homozygous mutations in <i>REC114</i> cause female infertility characterised by multiple pronuclei formation and early embryonic arrest
<i>NEK11</i> as a candidate high-penetrance melanoma susceptibility gene Dutch family
Cornelia de Lange syndrome: from molecular diagnosis to therapeutic approach
Novel phenotypes observed in patients with<i>ETV6</i>-linked leukaemia/familial thrombocytopenia syndrome and a biallelic<i>ARID5B</i>risk allele as leukaemogenic cofactor
Intronic<i>SMCHD1</i>variants in FSHD: testing the potential for CRISPR-Cas9 genome editing
Disruption of normal patterns of FOXF1 expression in a lethal disorder of lung development
Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy
Risk of metastatic pheochromocytoma and paraganglioma in <i>SDHx</i> mutation carriers: a systematic review and updated meta-analysis
Review of the scientific evolution of gene therapy for the treatment of homozygous familial hypercholesterolaemia: past, present and future perspectives
Gastric polyposis and desmoid tumours as a new familial adenomatous polyposis clinical variant associated with <i>APC</i> mutation at the extreme 3′-end
Increasing knowledge in <i>IGF1R</i> defects: lessons from 35 new patients
Complete loss of KCNA1 activity causes neonatal epileptic encephalopathy and dyskinesia
Optimised molecular genetic diagnostics of Fanconi anaemia by whole exome sequencing and functional studies
Recurrent homozygous damaging mutation in <i>TMX2</i>, encoding a protein disulfide isomerase, in four families with microlissencephaly
Presence of pathogenic copy number variants (CNVs) is correlated with socioeconomic status
Rapid, proteomic urine assay for monitoring progressive organ disease in Fabry disease
Impact of DNA source on genetic variant detection from human whole-genome sequencing data
Distal hereditary motor neuronopathy of the Jerash type is caused by a novel <i>SIGMAR1</i> c.500A&gt;T missense mutation
Germline <i>BRCA1</i> and <i>BRCA2</i> testing for breast cancer survivors
Enrichment of damaging missense variants in genes related with axonal guidance signalling in sporadic Meniere’s disease Spanish
High-sensitivity microsatellite instability assessment for the detection of mismatch repair defects in normal tissue of biallelic germline mismatch repair mutation carriers
Homozygous damaging SOD2 variant causes lethal neonatal dilated cardiomyopathy
Single-molecule optical mapping enables quantitative measurement of D4Z4 repeats in facioscapulohumeral muscular dystrophy (FSHD)
Myoclonic epilepsy, parkinsonism, schizophrenia and left-handedness as common neuropsychiatric features in 22q11.2 deletion syndrome
Paediatric systemic lupus erythematosus as a manifestation of constitutional mismatch repair deficiency
Biallelic mutations in<i>CFAP65</i>cause male infertility with multiple morphological abnormalities of the sperm flagella in humans and mice Han Chinese; Tunisian; Iranian ancestries; various human populations
Genetic aetiology of early infant deaths in a neonatal intensive care unit
Variant type is associated with disease characteristics in SDHB, SDHC and SDHD-linked phaeochromocytoma–paraganglioma
Predictability and inconsistencies of cognitive outcome in patients with phenylketonuria and personalised therapy: the challenge for the future guidelines
Open questions on the nature of Parkinson’s disease: from triggers to spreading pathology
Differential disruption of autoinhibition and defect in assembly of cytoskeleton during cell division decide the fate of human<i>DIAPH1</i>-related cytoskeletopathy
Estimating the effect size of the 15Q11.2 BP1–BP2 deletion and its contribution to neurodevelopmental symptoms: recommendations for practice
Postzygotic mosaicism in cerebral cavernous malformation
<i>De novo</i> SCAMP5 mutation causes a neurodevelopmental disorder with autistic features and seizures
<i>De novo</i> variants in <i>SLC12A6</i> cause sporadic early-onset progressive sensorimotor neuropathy
Long-read sequencing and haplotype linkage analysis enabled preimplantation genetic testing for patients carrying pathogenic inversions
Biallelic mutations in<i>CFAP65</i>lead to severe asthenoteratospermia due to acrosome hypoplasia and flagellum malformations Chinese
Genetic factors contributing to autism spectrum disorder in Williams-Beuren syndrome
Long-read sequencing identified repeat expansions in the 5′UTR of the<i>NOTCH2NLC</i>gene from Chinese patients with neuronal intranuclear inclusion disease Chinese; Japanese
‘Metaphyseal dysplasia without hypotrichosis’ can present with late-onset extraskeletal manifestations
Hypolipidaemia among patients with PMM2-CDG is associated with low circulating PCSK9 levels: a case report followed by observational and experimental studies
Tumour characteristics provide evidence for germline mismatch repair missense variant pathogenicity
Truncating mutations in exons 20 and 21 of<i>OFD1</i>can cause primary ciliary dyskinesia without associated syndromic symptoms Polish
Segregation of two variants suggests the presence of autosomal dominant and recessive forms of <i>WFS1</i>-related disease within the same family: expanding the phenotypic spectrum of Wolfram Syndrome
<i>RASA1</i>mosaic mutations in patients with capillary malformation-arteriovenous malformation
CCMG practice guideline: laboratory guidelines for next-generation sequencing
Reasons for and time to retraction of genetics articles published between 1970 and 2018
One in three highly selected Greek patients with breast cancer carries a loss-of-function variant in a cancer susceptibility gene
Clinical features and cancer risk in families with pathogenic<i>CDH1</i>variants irrespective of clinical criteria
SMCHD1 mutation spectrum for facioscapulohumeral muscular dystrophy type 2 (FSHD2) and Bosma arhinia microphthalmia syndrome (BAMS) reveals disease-specific localisation of variants in the ATPase doma
<i>JMG</i>in 2019: looking forward, looking back
Genome-wide association study identifies seven novel loci associating with circulating cytokines and cell adhesion molecules in Finns Finns
Genetic T-type calcium channelopathies
Genetic characteristics of retinitis pigmentosa in 1204 Japanese patients Japanese; East Asian; non-East Asian; East Asian-specific; Japanese population
<i>TNFRSF21</i> mutations cause high myopia Chinese (uses phrases 'A large Chinese family' and 'Chinese population')
New insights into 5α-reductase type 2 deficiency based on a multi-centre study: regional distribution and genotype–phenotype profiling of<i>SRD5A2</i>in 190 Chinese patients
Addition of a 161-SNP polygenic risk score to family history-based risk prediction: impact on clinical management in non-<i>BRCA1/2</i>breast cancer families
Familial bilateral cryptorchidism is caused by recessive variants in<i>RXFP2</i>
Loss-of-function mutations in SPEF2 cause multiple morphological abnormalities of the sperm flagella (MMAF)
Pathogenic variants in <i>PLOD3</i> result in a Stickler syndrome-like connective tissue disorder with vascular complications
Gastric cancer in Lynch syndrome is associated with underlying immune gastritis
Homozygous mutations in<i>SPEF2</i>induce multiple morphological abnormalities of the sperm flagella and male infertility Han Chinese; Iranian
Prenatal ultrasound findings of rasopathies in a cohort of 424 fetuses: update on genetic testing in the NGS era
Prevalence of germline pathogenic <i>BRCA1/2</i> variants in sequential epithelial ovarian cancer cases
Case for genome sequencing in infants and children with rare, undiagnosed or genetic diseases
Structural Aberrations with Secondary Implications (SASIs): consensus recommendations for reporting of cancer susceptibility genes identified during analysis of Copy Number Variants (CNVs)
Functional analysis of novel desert hedgehog gene variants improves the clinical interpretation of genomic data and provides a more accurate diagnosis for patients with 46,XY differences of sex develo
Bi-allelic loss of function variants of<i>TBX6</i>causes a spectrum of malformation of spine and rib including congenital scoliosis and spondylocostal dysostosis
Deciphering the complexity of the 4q and 10q subtelomeres by molecular combing in healthy individuals and patients with facioscapulohumeral dystrophy
Mutation-specific Fabry disease patient-derived cell model to evaluate the amenability to chaperone therapy
A novel mutation in the<i>GFAP</i>gene expands the phenotype of Alexander disease
Multivariate genome-wide association study of rapid automatised naming and rapid alternating stimulus in Hispanic American and African–American youth children of European ancestry; Hispanic American; African–American
Resectable lung lesions malignancy assessment and cancer detection by ultra-deep sequencing of targeted gene mutations in plasma cell-free DNA
Deleterious somatic variants in 473 consecutive individuals with ovarian cancer: results of the observational AGO-TR1 study (NCT02222883)
Towards controlled terminology for reporting germline cancer susceptibility variants: an ENIGMA report
Whole genome paired-end sequencing elucidates functional and phenotypic consequences of balanced chromosomal rearrangement in patients with developmental disorders
Pathogenic variants in the AFG3L2 proteolytic domain cause SCA28 through haploinsufficiency and proteostatic stress-driven OMA1 activation
Allele frequency analysis of variants reported to cause autosomal dominant inherited retinal diseases question the involvement of 19% of genes and 10% of reported pathogenic variants
Genetic linkage analysis of a large family identifies <i>FIGN</i> as a candidate modulator of reduced penetrance in heritable pulmonary arterial hypertension Iberian; Iberian family
Alternative splicing and ACMG-AMP-2015-based classification of PALB2 genetic variants: an ENIGMA report
Targeted next-generation sequencing detects rare genetic events in pheochromocytoma and paraganglioma
Clinical characteristics of patients with colorectal cancer with double somatic mismatch repair mutations compared with Lynch syndrome
Mutations in <i>NLRP2</i> and <i>NLRP5</i> cause female infertility characterised by early embryonic arrest
Evidence for heightened genetic instability in precancerous spasmolytic polypeptide expressing gastric glands
Promising member of the short interspersed nuclear elements (<i>Alu</i> elements): mechanisms and clinical applications in human cancers
Advances in identification of genes involved in autosomal recessive intellectual disability: a brief review
Genetic landscape of Rett syndrome-like phenotypes revealed by whole exome sequencing
<i>NAA10</i> polyadenylation signal variants cause syndromic microphthalmia
Parkinson’s disease GWAS-linked Park16 carriers show greater motor progression
Genotype–phenotype correlations in ataxia telangiectasia patients with <i>ATM</i> c.3576G&gt;A and c.8147T&gt;C mutations
Genome-wide meta-analysis identifies genetic locus on chromosome 9 associated with Modic changes
Distal chromosome 16p11.2 duplications containing <i>SH2B1</i> in patients with scoliosis
Contribution of spurious transcription to intellectual disability disorders
Associations of CDH1 germline variant location and cancer phenotype in families with hereditary diffuse gastric cancer (HDGC)
Natural history of renal tumours in von Hippel-Lindau disease: a large retrospective study of Chinese patients
Genetic diagnosis of subfertility: the impact of meiosis and maternal effects
<i>H2AFY</i> promoter deletion causes <i>PITX1</i> endoactivation and Liebenberg syndrome
Features, genetics and their correlation in Jalili syndrome: a systematic review
Diagnosis of ‘possible’ mitochondrial disease: an existential crisis
<i>SMAD3</i>pathogenic variants: risk for thoracic aortic disease and associated complications from the Montalcino Aortic Consortium
Clinical spectrum and pleiotropic nature of<i>CDH1</i>germline mutations