Journal of Medical Genetics - 2024

131 articles | Last updated: 2025-12-03 14:12:57
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T A T A T A T A
Reassessment and reclassification of variants of unknown significance in patients with cardiomyopathy in a specialist department
Faecal incontinence disorders in Wolfram syndrome: a new manifestation
Validation of the NCCN/Yale criteria for the identification of<i>CDH1</i>pathogenic variant carriers European Reference Network on Genetic Tumour Risk Syndromes; European cohort of CDH1 PV carriers
Hiding in plain sight: a partial deletion of<i>BRCA1</i>exon 7 undetectable by MLPA is a Nepali founder variant
Unveiling non-coding<i>DMD</i>variants: synergising RNA sequencing and DNA sequencing for enhanced molecular diagnosis
Predicting the likelihood of<i>BRCA1/2</i>germline pathogenic variants in unselected patients with breast cancer: analysis of more than 10,000 individuals
Aortic and arterial manifestations and clinical features in<i>TGFB3</i>-related heritable thoracic aortic disease: results from the Montalcino Aortic Consortium
Retrospective study on the utility of optical genome mapping as a follow-up method in genetic diagnostics
Genomic sequencing in paediatric oncology: navigating conflicting roles and responsibilities
Tumour spectrum in<i>AKT1</i>-related Proteus syndrome: a systematic review of clinical reports and series
Six at Sixty. Commentary on identification of the<i>PTEN</i>gene as a major contributor to autism spectrum disorder
<i>KIF21A</i>-associated peripheral neuropathy defined by impaired binding with TUBB3
Portraying the full picture of Neurofibromatosis–Noonan syndrome: a systematic review of literature
Novel<i>HYLS1</i>variants associated with Joubert syndrome suggest potential genotype-phenotype correlates
National survey on the prevalence of single-gene aetiologies for genetic developmental and epileptic encephalopathies in Italy a European country
Enhancing variant of uncertain significance (VUS) interpretation in neurogenetics: collaborative experiences from a tertiary care centre
Long-read sequencing for detection and subtyping of Prader-Willi and Angelman syndromes
Heterozygous de novo variants in<i>HSPD1</i>cause hypomyelinating leukodystrophy through impaired HSP60 oligomerisation
Clinical and mutational signatures of<i>CRB1</i>-associated retinopathies: a multicentre study
Six at Sixty. Malignant peripheral nerve sheath tumours in NF1: 20-year review of a highly cited paper
Accurate prenatal diagnosis of facioscapulohumeral muscular dystrophy 1 using nanopore sequencing
Cardiac rhabdomyoma: a rare feature of Birt-Hogg-Dubé syndrome
<i>WDR45</i>variants as a major cause for a clinically variable intellectual disability syndrome from early infancy in females
Lynch syndrome diagnostic testing pathways in endometrial cancers: a nationwide English registry-based study
Authors’ response to the commentary by Kivela<i>et al</i>on Hany<i>et al</i>(2024)
Commentary on<i>Estimating cancer risk in carriers of Lynch syndrome variants in UK Biobank</i>
Canadian College of Medical Geneticists: clinical practice advisory document – responsibility to recontact for reinterpretation of clinical genetic testing
<i>UBTF</i>haploinsufficiency associated with<i>UBTF</i>-related global developmental delay and distinctive facial features without neuroregression
K acetyltransferase 2B (<i>KAT2B</i>) variants can be responsible for early onset steroid-resistant nephrotic syndrome
Classification of<i>PTEN</i>germline non-truncating variants: a new approach to interpretation
Novel variants and genotype-phenotype correlation in a multicentre cohort of GNE myopathy in China Chinese, Japanese, Korean, Jewish
Pathogenic <i>SATB2</i> missense variants affecting p.Gly392 have variable functional implications and result in diverse clinical phenotypes
Histone modifications in Duchenne muscular dystrophy: pathogenesis insights and therapeutic implications
Rare disease genomic testing in the UK and Ireland: promoting timely and equitable access
Male–female phenotype correlation and dissociation related to mutations in the <i>ARX</i> gene
Novel germline <i>TP53</i> variant (p.(Phe109Ile)) confers high risk of cancer
A novel pathogenic germline chromosome 3 inversion in von Hippel-Lindau disease
Christianson syndrome across the lifespan: genetic mutations and longitudinal study in children, adolescents, and adults
The PS4-likelihood ratio calculator: flexible allocation of evidence weighting for case-control data in variant classification
Biallelic variants in α-tubulin isotypes cause female infertility characterised as recurrent preimplantation embryo arrest
Genetic findings in people with schwannomas who do not meet clinical diagnostic criteria for <i>NF2</i> -related schwannomatosis
Heterozygous <i>COL17A1</i> variants are a frequent cause of amelogenesis imperfecta
Protein-truncating and rare missense variants in <i>ATM</i> and <i>CHEK2</i> and associations with cancer in UK Biobank whole-exome sequence data
Loss-of-function variants in <i>JPH1</i> cause congenital myopathy with prominent facial and ocular involvement
Splice site variants in the canonical donor site of <i>MED13L</i> exon 7 lead to intron retention in patients with <i>MED13L</i> syndrome
<i>JMG</i>at 60
Shared germline genomic variants in two patients with double primary gastrointestinal stromal tumours (GISTs)
From onset to blindness: a comprehensive analysis of <i>RPGR</i> -associated X-linked retinopathy in a large cohort in China
Genetics of prostate cancer: a review of latest evidence PwPs of African descent; ethnicity
Genotype–phenotype correlation of <i>SQSTM1</i> variants in patients with amyotrophic lateral sclerosis Chinese
Novel truncating germline variant reinforces <i>TINF2</i> as a susceptibility gene for familial non-medullary thyroid cancer
Is renal cell carcinoma associated with <i>MITF</i> c.952G&gt;A (p.E318K)?
Double gonosomal mosaicism as an unusual hereditary mechanism in familial <i>GRIN2A</i> -related disorder
Developmental dysplasia of the hip caused by homozygous <i>TRIM33</i> pathogenic variant affecting downstream BMP pathway Bedouin
Germline testing for breast cancer patients in England: illogical to prioritise grade 1 breast cancer aged 30–39 over grade 3 aged 40–49 years?
Estimating cancer risk in carriers of Lynch syndrome variants in UK Biobank
Development and internal validation of a clinical risk score to predict incident renal and pulmonary tumours in people with tuberous sclerosis complex
<i>IFIH1</i> variants are associated with generalised epilepsy preceded by febrile seizures
Long-read sequencing identifies an SVA_D retrotransposon insertion deep within the intron of<i>ATP7A</i>as a novel cause of occipital horn syndrome
Arterial aneurysm and dissection: toward the evolving phenotype of Tatton-Brown-Rahman syndrome
Large<i>TRAPPC11</i>gene deletions as a cause of muscular dystrophy and their estimated genesis
Expanding the genetic and clinical spectrum of Tatton-Brown-Rahman syndrome in a series of 24 French patients
<i>NF2</i>-related schwannomatosis and other schwannomatosis: an updated genetic and epidemiological study
Amyotrophic lateral sclerosis patients with various gene mutations show diverse motor phenotypes and survival in China
Complex structural variation and nonsense variant<i>in trans</i>cause<i>VPS50</i>-related disorder
Next-generation sequencing in Charcot-Marie-Tooth: a proposal for improvement of ACMG guidelines for variant evaluation
Extending the clinical spectrum of X-linked Tonne-Kalscheuer syndrome (TOKAS): new insights from the fetal perspective
<i>ZFHX3</i> variants cause childhood partial epilepsy and infantile spasms with favourable outcomes
Disease-specific variant interpretation highlighted the genetic findings in 2325 Japanese patients with retinitis pigmentosa and allied diseases
Encephalocraniocutaneous lipomatosis phenotype associated with mosaic biallelic pathogenic variants in the<i>NF1</i>gene
Guidelines for NGS procedures applied to prenatal diagnosis by the Spanish Society of Gynecology and Obstetrics and the Spanish Association of Prenatal Diagnosis
Validation of the BOADICEA model in a prospective cohort of<i>BRCA1/2</i>pathogenic variant carriers
Exome sequencing of 1190 non-syndromic clubfoot cases reveals <i>HOXD12</i> as a novel disease gene "European ancestry"; "non-European ancestry"
ZFYVE19 deficiency: a ciliopathy involving failure of cell division, with cell death
Carrier testing for partners of<i>MUTYH</i>variant carriers: UK Cancer Genetics Group recommendations
Variant classification changes over time in the clinical molecular diagnostic laboratory setting
Comprehensive preimplantation genetic testing for balanced insertional translocation carriers
Rare variant association analyses reveal the significant contribution of carbohydrate metabolic disturbance in severe adolescent idiopathic scoliosis Han Chinese
<i>POT1</i>and multiple primary melanomas: the dermatological phenotype European ancestry
Comprehensive genomic filtering algorithm to expose the cause of skewed X chromosome inactivation. The proof of concept in female haemophilia expression
Expanded targeted preconception screening panel in Israel: findings and insights "ethnically-based screening tests", "uniquely diverse Israeli population", references to "Israeli" p
Challenges in developing and implementing international best practice guidance for intermediate-risk variants in cancer susceptibility genes:<i>APC</i>c.3920T&gt;A p.(Ile1307Lys) as an exemplar
Homozygous variant in<i>TKFC</i>abolishing triokinase activities is associated with isolated immunodeficiency
Genetic evidence for splicing-dependent structural and functional plasticity in CASK protein
Novel mutation leading to splice donor loss in a conserved site of<i>DMD</i>gene causes Duchenne muscular dystrophy with cryptorchidism Chinese
Systematic reanalysis of copy number losses of uncertain clinical significance
Pathogenic variant detection rate varies considerably in male breast cancer families and sporadic cases: minimal additional contribution beyond<i>BRCA2, BRCA1</i>and<i>CHEK2</i>
Response to commentary: Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III
<i>BRCA</i> awareness and testing experience in the UK Jewish population: a qualitative study UK Jewish; Jewish; Jewish people; Jewish community
Phenotypic characterisation of<i>SMAD4</i>variant carriers
Comment to: Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE stud
Variant characterisation and clinical profile in a large cohort of patients with Ellis-van Creveld syndrome and a family with Weyers acrofacial dysostosis
Extent of investigation and management of cases of ‘unexplained’ mismatch repair deficiency (u-dMMR): a UK Cancer Genetics Group consensus
Subcellular localisation of truncated MAGEL2 proteins: insight into the molecular pathology of Schaaf-Yang syndrome
A comparative medical genomics approach may facilitate the interpretation of rare missense variation
Diagnosis and management in Rubinstein-Taybi syndrome: first international consensus statement
Cerebral visual impairment: genetic diagnoses and phenotypic associations
Pathogenic variants affecting the TB5 domain of the fibrillin-1 protein: not only in geleophysic/acromicric dysplasias but also in Marfan syndrome
Biallelic variants in Plexin B2 ( <i>PLXNB2</i> ) cause amelogenesis imperfecta, hearing loss and intellectual disability
Survey of service needs to embed genome sequencing for motor neuron disease in neurology in the English National Health Service
Dissecting genetic architecture of rare dystonia: genetic, molecular and clinical insights populations of European descent
Comparing the frequency of variants of uncertain significance (VUS) between ancestry groups in a paediatric epilepsy cohort non-European populations
<i>NASP</i> gene contributes to autism by epigenetic dysregulation of neural and immune pathways Chinese
Novel <i>TUBA4A</i> variant causes congenital myopathy with focal myofibrillar disorganisation
Skewed X-chromosome inactivation drives the proportion of<i>DNAAF6</i>-defective airway motile cilia and variable expressivity in primary ciliary dyskinesia
Exploring the molecular pathways linking sleep phenotypes and<i>POGZ</i>-associated neurodevelopmental disorder
Bi-allelic variants in chromatoid body protein TDRD6 cause spermiogenesis defects and severe oligoasthenoteratozoospermia in humans Chinese
Improved sensitivity for detection of pathogenic variants in familial<i>NF2</i>-related schwannomatosis
<i>ZNF142</i>mutation causes sex-dependent neurologic disorder Bedouin kindred
Variant reclassification and clinical implications ancestry (as in 'ancestry studies')
Molecular diagnosis, clinical evaluation and phenotypic spectrum of Townes-Brocks syndrome: insights from a large Chinese hearing loss cohort
Childhood-onset hypertrophic cardiomyopathy caused by thin-filament sarcomeric variants
Heterozygous deletion of<i>HOXC10-HOXC9</i>causes lower limb abnormalities in congenital vertical talus
BTB domain mutations perturbing KCTD15 oligomerisation cause a distinctive frontonasal dysplasia syndrome
Expanding the phenotype of Kleefstra syndrome: speech, language and cognition in 103 individuals
Mosaic RASopathies concept: different skin lesions, same systemic manifestations?
Homozygous<i>SMAD6</i>variants in two unrelated patients with craniosynostosis and radioulnar synostosis
De novo heterozygous missense variants in<i>CELSR1</i>as cause of fetal pleural effusions and progressive fetal hydrops
Genotype and phenotype correlation of<i>PHACTR1</i>-related neurological disorders
Whole-exome sequencing reveals causative genetic variants for several overgrowth syndromes in molecularly negative Beckwith-Wiedemann spectrum
<i>GRN</i>mutation spectrum and genotype–phenotype correlation in Chinese dementia patients: data from PUMCH dementia cohort
Iron and risk of dementia: Mendelian randomisation analysis in UK Biobank European (N=451284); European groups; participants clustered into European
Charis Eng: an appreciation
<i>JMG</i>in 2025
Advances in research on the mechanism of tsRNA action in tumours
Clonazepam repurposing in<i>ARID1B</i>patients through conventional RCT and N-of-1 trials: an experimental strategy for orphan disease development
Congenital urinary tract anomalies are a variable finding associated with nevoid basal cell carcinoma syndrome
Cystic fibrosis carrier screening in Australia: comparing sequencing and targeted panels across diverse ancestries non-European ancestry groups; Northern Europe group; Southern Asia group
<i>TFAP2E</i>is implicated in central nervous system, orofacial and maxillofacial anomalies
A tandem duplication of exon 42 of the<i>DMD</i>gene is a likely benign variant
The NHS England Jewish BRCA Testing Programme: overview after first year of implementation (2023–2024)