Clinical Genetics - 2021

203 articles | Last updated: 2025-12-03 14:12:56
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Clinical characterization of novel <scp>HSPA9</scp> splice variant in a Chinese woman
Multi‐centre study of the clinical features and gene variant spectrum of Gitelman syndrome in Chinese children
Growth, development, and phenotypic spectrum of individuals with deletions of 2q33.1 involving <i>SATB2</i>
Clinical presentation and molecular characterization of a novel patient with variant <i><scp>POC1A</scp>‐</i>related syndrome
A rare disease and education: Neurofibromatosis type 1 decreases educational attainment
<scp><i>IQSEC2</i></scp> disorder: A new disease entity or a Rett spectrum continuum?
<scp>Smith‐Magenis</scp> syndrome: Clinical and behavioral characteristics in a large retrospective cohort 47 European patients
Biallelic loss‐of‐function <scp><i>HACD1</i></scp> variants are a bona fide cause of congenital myopathy
Compound heterozygosity for <scp>PTPN11</scp> variants in a subject with Noonan syndrome provides insights into the mechanism of <scp>SHP2</scp>‐related disorders
Phenotypic spectrum of the <scp>RBM10</scp>‐mediated intellectual disability and congenital malformation syndrome beyond classic <scp>TARP</scp> syndrome features
Genetic diagnosis of infantile‐onset epilepsy in the clinic: Application of whole‐exome sequencing following epilepsy gene panel testing
Tooth agenesis: What do we know and is there a connection to cancer?
Null variants in <scp><i>DYSF</i></scp> result in earlier symptom onset
Genetic variations and clinical spectrum of dystroglycanopathy in a large cohort of Chinese patients
A novel heterozygous variant in <scp><i>FGF9</i></scp> associated with previously unreported features of multiple synostosis syndrome 3
Duane retraction syndrome characterized by inner ear agenesis and neurodevelopmental phenotype in an Italian family with a variant in <scp><i>MAFB</i></scp> Italian family
Factors influencing genetic counseling and testing for hereditary breast and ovarian cancer syndrome in a large <scp>US</scp> health care system
Challenging the traditional approach for interpreting genetic variants: Lessons from Fabry disease
Rare and de novo duplications containing <scp><i>TCF20</i></scp> are associated with a neurodevelopmental disorder
Utility of clinical exome sequencing in progressive myoclonus epilepsy syndromes: An exploratory analysis
Embryonal sarcoma of the liver in a girl with Cockayne syndrome
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Neurodevelopmental phenotype in 36 new patients with 8p inverted duplication–deletion: Genotype–phenotype correlation for anomalies of the corpus callosum
A homozygous <scp><i>ABHD16A</i></scp> variant causes a complex hereditary spastic paraplegia with developmental delay, absent speech, and characteristic face
Segmental undergrowth is associated with pathogenic variants in vascular malformation genes: A retrospective case‐series study
Mitochondrial developmental encephalopathy with bilateral optic neuropathy related to homozygous variants in<scp><i>IMMT</i></scp>gene
Early psychosis in <scp>Thauvin‐Robinet‐Faivre</scp> syndrome, a complication of the disease?
<scp><i>DYRK1B</i></scp> haploinsufficiency in a family with metabolic syndrome and abnormal cognition
Bi‐allelic variants in <scp><i>MDH2</i></scp>: Expanding the clinical phenotype
Identifying biomarkers for prenatal diagnosis of neural tube defects based on “omics”
<scp><i>TAB2</i></scp> variants cause cardiovascular heart disease, connective tissue disorder, and developmental delay
Expanding the spectrum of syndromic <i>PPP2R3C</i>‐related <scp>XY</scp> gonadal dysgenesis to <scp>XX</scp> gonadal dysgenesis Turkish and Indian descent
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Expanding the genotypic spectrum of <scp><i>TXNL4A</i></scp> variants in <scp>Burn‐McKeown</scp> syndrome
Biallelic <scp><i>ZNFX1</i></scp> variants are associated with a spectrum of immuno‐hematological abnormalities
Kosaki overgrowth syndrome due to a novel de novo <scp><i>PDGFRB</i></scp> variant
Mono‐allelic loss of <scp><i>YTHDF3</i></scp> and neurodevelopmental disorder: clinical features of four individuals with 8q12.3 deletions
A <scp><i>SPIDR</i></scp> homozygous nonsense pathogenic variant in isolated primary ovarian insufficiency with chromosomal instability Indian
First case series of Polish patients with cerebrotendinous xanthomatosis and systematic review of cases from the 21st century "Polish patients", "Polish and Asian populations", "Asian and non-Asian populations"
<i>In silico</i> genome‐wide gene‐based association analysis reveals new genes predisposing to coronary artery disease
Clinical utility of liquid biopsy in breast cancer: A systematic review
The broader phenotypic spectrum of congenital caudal abnormalities associated with mutations in the caudal type homeobox 2 gene
Use of medical exome sequencing for identification of underlying genetic defects in <scp>NICU</scp>: Experience in a cohort of 2303 neonates in China
Copy number variations in a Brazilian cohort with autism spectrum disorders highlight the contribution of cell adhesion genes "144 Brazilian individuals with ASD of strong European and African ancestries."; "144 Brazilian individuals with ASD of strong European and African ancestries."
Genetic and metabolic profiling of individuals with <scp>Phelan‐McDermid</scp> syndrome presenting with seizures
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Biallelic <scp><i>TMEM260</i></scp> variants cause truncus arteriosus, with or without renal defects
Prenatal exome sequencing: A useful tool for the fetal neurologist
A homozygous loss‐of‐function mutation in <scp><i>FBXO43</i></scp> causes human non‐obstructive azoospermia Chinese
Dominant‐negative pathogenic variant <scp>BRIP1</scp> c.<scp>1045G</scp>&gt;C is a high‐risk allele for non‐mucinous epithelial ovarian cancer: A case‐control study
Correlation of <scp>DUOX2</scp> residual enzymatic activity with phenotype in congenital hypothyroidism caused by biallelic <scp><i>DUOX2</i></scp> defects
Pathogenic variants in the survival of motor neurons complex gene <scp><i>GEMIN5</i></scp> cause cerebellar atrophy
Bi‐allelic mutations in <scp><i>MCIDA</i>S</scp> and <scp><i>CCNO</i></scp> cause human infertility associated with abnormal gamete transport
<scp><i>RNF43</i></scp> pathogenic Germline variant in a family with colorectal cancer
<scp><i>HK1</i></scp> haemolytic anaemia in association with a neurological phenotype and co‐existing <scp><i>CEP290</i> Meckel–Gruber</scp> in a Romani family Romani family
Identification and molecular analysis of 17 novel variants of hydroxymethylbilane synthase in Chinese patients with acute intermittent porphyria
Novel bi‐allelic <scp><i>HPDL</i></scp> variants cause hereditary spastic paraplegia in a Chinese patient
The Thai reference exome (T‐REx) variant database non‐European ancestry; Thai population; East and Southeast Asian populations; Thai subpopulations; ethno‐geographic groups;
<i>GLI3</i> variants causing isolated polysyndactyly are not restricted to the protein's C‐terminal third
The clinical efficacy and safety of <scp>anti‐IgE</scp> therapy in recessive dystrophic epidermolysis bullosa
Prevalence and clinical prediction of mitochondrial disorders in a large neuropediatric cohort
Diagnostic yield of genetic testing in 324 infants with hypotonia
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Novel <scp><i>KMT2B</i></scp> mutation causes cerebellar ataxia: Expanding the clinical phenotype
Hypomorphic alleles pose challenges in rare disease genomic variant interpretation
Clinical, genetic, and histological features of centronuclear myopathy in the Netherlands
Homozygous <scp><i>TRAPPC11</i></scp> truncating variant revealing segmental uniparental disomy of chromosome 4 as a cause of a recessive limb‐girdle muscular dystrophy‐18
A <scp>CELSR1</scp> variant in a patient with pulmonary arterial hypertension
A novel <i>POF1B</i> variant in a Chinese patient is associated with premature ovarian failure
Bi‐allelic loss of function variants in <scp>GOLGA2</scp> are associated with a complex neurological phenotype: Report of a second family
Genetic counseling and testing for hereditary hemorrhagic telangiectasia
Clinical and molecular delineation of <scp><i>PUS3</i></scp>‐associated neurodevelopmental disorders
Molecular autopsy by proxy in preconception counseling
Kaposiform hemangioendothelioma further broadens the phenotype of <scp>PIK3CA</scp>‐related overgrowth spectrum
A novel <scp><i>POU1F1</i></scp> pathogenic variant: Two familial case reports with phenotype expansion
Spectrum of neuro‐genetic disorders in the United Arab Emirates national population
Delineating the expanding phenotype of <scp><i>HERC2</i></scp>‐related disorders: The impact of biallelic loss of function versus missense variation
A new synonymous variant involving an <scp><i>mRNA</i></scp> splicing site in <scp><i>CYP21A2</i></scp> detected in 12 unrelated patients with deficiency of 21‐hydroxylase
Psychosocial interventions and needs among individuals and families with <scp>Li‐Fraumeni</scp> syndrome: A scoping review
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Clinical variability of neurofibromatosis 1: A modifying role of cooccurring <scp><i>PTPN11</i></scp> variants and atypical brain <scp>MRI</scp> findings
Congenital limb deficiency: Genetic investigation of 44 individuals presenting mainly longitudinal defects in isolated or syndromic forms African ancestry
Clinical and genetic spectrum of 104 Indian families with central nervous system white matter abnormalities Indian individuals; our population
Whole genome sequencing identifies rare germline variants enriched in cancer related genes in first degree relatives of familial pancreatic cancer patients
A C‐terminal <scp><i>BCOR</i></scp> nonsense variant in a male patient expands the phenotypic spectrum of <scp><i>BCOR</i></scp>‐associated syndromic microphthalmia
Genetic spectrum of familial hypercholesterolemia and correlations with clinical expression: Implications for diagnosis improvement
Facioscapulohumeral muscular dystrophy—Reproductive counseling, pregnancy, and delivery in a complex multigenetic disease
The landscape of autosomal recessive variants in an isolated community: Implications for population screening for reproductive purposes
Epileptic encephalopathy caused by <scp>ARV1</scp> deficiency: Refinement of the genotype–phenotype spectrum and functional impact on <scp>GPI</scp>‐anchored proteins
Short stature with low insulin‐like growth factor 1 availability due to pregnancy‐associated plasma protein <scp>A2</scp> deficiency in a Saudi family
A 24‐generation‐old founder mutation impairs splicing of <scp><i>RBBP8</i></scp> in Pakistani families affected with Jawad syndrome
Exome sequencing of familial adenomatous polyposis‐like individuals identifies both known and novel causative genes
A case of <scp><i>ASAH1</i></scp>‐related pure SMA evolving into adult‐onset Farber disease
A standard of care for individuals with <scp><i>PIK3CA</i></scp>‐related disorders: An international expert consensus statement
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Hypermobile <scp>Ehlers–Danlos</scp> syndrome: A review and a critical appraisal of published genetic research to date
Knowledge, views and expectations for cancer polygenic risk testing in clinical practice: A cross‐sectional survey of health professionals
<scp><i>ZMYND11</i></scp> variants are a novel cause of centrotemporal and generalised epilepsies with neurodevelopmental disorder
“I have to start learning how to live with becoming sick”: A scoping review of the lived experiences of people with Huntington's disease
<scp>CHEDDA</scp> syndrome is an underrecognized neurodevelopmental disorder with a highly restricted <scp><i>ATN1</i></scp> mutation spectrum
The first two <scp>non‐Finnish <i>HYLS1</i></scp> variants: Expanding the phenotypic spectrum of hydrolethalus syndrome Finnish; non-Finnish
<scp>KRAS</scp> mutation identified in a patient with melorheostosis and extended lymphangiomatosis treated with sirolimus and trametinib
Recalling the pathology of Parkinson's disease; lacking exact figure of prevalence and genetic evidence in Asia with an alarming outcome: A time to step‐up
Tenorio syndrome: Description of 14 novel cases and review of the clinical and molecular features
<scp>EPHA7</scp> haploinsufficiency is associated with a neurodevelopmental disorder
De novo pathogenic <scp><i>DHX30</i></scp> variants in two cases
Expanding the phenotype of <scp><i>SETD5</i></scp>‐related disorder and presenting a novel association with bone fragility
Heterozygous <scp><i>HMGB1</i></scp> loss‐of‐function variants are associated with developmental delay and microcephaly
Expanding the phenotypic spectrum of FINCA (fibrosis, neurodegeneration, and cerebral angiomatosis) syndrome beyond infancy
Genotypic and phenotypic analysis in 51 Chinese patients with primary distal renal tubular acidosis Europeans; Chinese populations
How to deal with uncertainty in prenatal genomics: A systematic review of guidelines and policies
Two cases of <scp><i>DCDC2</i></scp>‐related neonatal sclerosing cholangitis with developmental delay and literature review
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Novel <scp><i>CDK10</i></scp> variants with multicystic dysplastic kidney, left ventricular non‐compaction, and a solitary median maxillary central incisor
<scp><i>PKD2</i></scp> gene variants in Chinese patients with autosomal dominant polycystic kidney disease
Patients with acephalic spermatozoa syndrome linked to novel <i>TSGA10/PMFBP1</i> variants have favorable pregnancy outcomes from intracytoplasmic sperm injection
Biallelic mutations in <scp><i>KATNAL2</i></scp> cause male infertility due to oligo‐astheno‐teratozoospermia
Out‐of‐pocket and private pay in clinical genetic testing: A scoping review
Lysosomal storage disorders as an etiology of nonimmune hydrops fetalis: A systematic review
Triploid pregnancy–Clinical implications
Bi‐allelic loss of <scp><i>ERGIC1</i></scp> causes relatively mild arthrogryposis
A retrospective analysis of growth hormone therapy in children with <scp>Schaaf–Yang</scp> syndrome
Inflammatory factors, <scp>genetic variants</scp>, and predisposition for preterm birth
Homozygous missense <scp><i>VPS16</i></scp> variant is associated with a novel disease, resembling mucopolysaccharidosis‐plus syndrome in two siblings
Isolated‐ and <scp>Beckwith‐Wiedemann</scp> syndrome related‐ lateralised overgrowth (hemihypertrophy): Clinical and molecular correlations in 94 individuals
Current and experimental therapeutics for Fabry disease pan-ethnic
The importance of precision medicine in modern molecular oncology
Copy number variation analysis implicates novel pathways in patients with oculo‐auriculo‐vertebral‐spectrum and congenital heart defects
Pathogenic variants of <i>ATG4D</i> in infertile men with non‐obstructive azoospermia identified using whole‐exome sequencing
Spinocerebellar ataxia type 2 from an evolutionary perspective: Systematic review and meta‐analysis
<scp><i>PPP3CA</i></scp> truncating variants clustered in the regulatory domain cause early‐onset refractory epilepsy
<scp>COG1‐</scp>congenital disorders of glycosylation: Milder presentation and review
A homozygous missense mutation in <scp><i>TBPL2</i></scp> is associated with oocyte maturation arrest and degeneration
<i>ANKRD11</i> variants: <scp>KBG</scp> syndrome and beyond
Molecular diagnosis for 55 fetuses with skeletal dysplasias by whole‐exome sequencing: A retrospective cohort study
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Nonsense‐mediated mRNA decay efficiency influences bleeding severity in <i>ITGA2B</i> c.<scp>2659C</scp> &gt; T (p.<scp>Q887X</scp>) knock‐in mice
Protein kinase <scp>D1</scp> variant associated with human epilepsy and peripheral nerve hypermyelination
Periodontal (formerly type <scp>VIII</scp>) <scp>Ehlers–Danlos</scp> syndrome: Description of 13 novel cases and expansion of the clinical phenotype
Biallelic start loss variant, c.<scp>1A</scp> &gt; G in <scp><i>GCSH</i></scp> is associated with variant nonketotic hyperglycinemia Indian
Severe congenital cutis laxa: Identification of novel homozygous <scp><i>LOX</i></scp> gene variants in two families
The genetic underpinnings of anthracycline‐induced cardiomyopathy predisposition
Molecular genetics of inherited retinal degenerations in Icelandic patients ancestral North-Western European nations
Clinical management among individuals with variant of uncertain significance in hereditary cancer: A systematic review and meta‐analysis
Zebrafish modeling mimics developmental phenotype of patients with <scp><i>RAPGEF1</i></scp> mutation
Rapid exome sequencing as the first‐tier investigation for diagnosis of acutely and severely ill children and adults in Thailand
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Pitfalls and challenges in genetic test interpretation: An exploration of genetic professionals experience with interpretation of results
Novel <scp><i>ANO5</i></scp> intronic Roma variant alters splicing causing muscular dystrophy Roma; Iberian Roma population
<i><scp>NKX2</scp>.1</i> run‐on mutation associated to familial brain–lung–thyroid syndrome
Biallelic <scp><i>RFC1</i></scp> pentanucleotide repeat expansions in Greek patients with late‐onset ataxia
<scp><i>DYNC2H1</i></scp> variants cause Leber congenital amaurosis without syndromic features
Third case of <scp>Bardet‐Biedl</scp> syndrome caused by a biallelic variant predicted to affect splicing of <scp><i>IFT74</i></scp>
Clinical and molecular delineation of spondylocostal dysostosis type 3
A novel bi‐allelic loss‐of‐function mutation in <scp><i>STIM1</i></scp> expands the phenotype of <scp>STIM1</scp>‐related diseases
Expansion of the clinical and molecular spectrum of an <scp>XPD</scp>‐related disorder linked to biallelic mutations in <scp><i>ERCC2</i></scp> gene
<scp><i>GCH1</i></scp> mutations in hereditary spastic paraplegia
Exome sequencing utility in defining the genetic landscape of hearing loss and novel‐gene discovery in Iran
Clinical insights and molecular study of three foetuses with <scp><i>DYNC2H1</i></scp> gene mutation causing short rib thoracic dystrophy
Featured Cover Chinese
Congenital ataxia due to novel variant in <scp><i>ATP8A2</i></scp>
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Genetic and phenotypic analysis of 101 patients with developmental delay or intellectual disability using whole‐exome sequencing
Mapping leprosy‐associated coding variants of interleukin genes by targeted sequencing
Keratoconus in a patient with <scp><i>B3GALT6</i></scp>‐related disorder
Heterozygous <scp><i>NOTCH1</i></scp> deletion associated with variable congenital heart defects
Otological manifestations in branchiootorenal spectrum disorder: A systematic review and meta‐analysis
<i>De novo</i> variants in neurodevelopmental disorders—experiences from a tertiary care center
Influence of molecular classes and growth hormone treatment on growth and dysmorphology in <scp>Prader‐Willi</scp> syndrome: A multicenter study
<scp>FARS1</scp>‐related disorders caused by bi‐allelic mutations in cytosolic phenylalanyl‐<scp>tRNA</scp> synthetase genes: Look beyond the lungs!
Improving clinical interpretation of five <scp><i>KRIT1</i></scp> and <scp><i>PDCD10</i></scp> intronic variants
Whole genome sequencing reveals biallelic <scp><i>PLA2G6</i></scp> mutations in siblings with cerebellar atrophy and cap myopathy
<scp>Health‐related</scp> quality of life in adults with osteogenesis imperfecta
Novel pathogenic variants in <scp><i>NLRP7</i></scp>, <scp><i>NLRP5</i>,</scp> and <scp><i>PADI6</i></scp> in patients with recurrent hydatidiform moles and reproductive failure
A rare case of fatty <scp>acyl‐CoA</scp> reductase 1 deficiency in an Indian infant manifesting rhizomelic chondrodystrophy phenotype
Immune deficiency, autoimmune disease and intellectual disability: A pleiotropic disorder caused by biallelic variants in the <scp><i>TPP2</i></scp> gene Swiss founder mutation
<scp><i>SMPD3</i>‐<i>ALK</i></scp>: A novel <scp><i>ALK</i></scp> fusion gene in lung adenocarcinoma
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Forty‐seven pathogenic variants causing autosomal recessive disorders are shared by Israeli and Saudi Arabian Arabs Arabs; Muslim Arabs; Bedouins; Bedouin origin; Saudi Peninsula; Israel; Saudi Arabia; populations
Accuracy diagnosis improvement of Fabry disease from dried blood spots: Enzyme activity, <scp>lyso‐Gb3</scp> accumulation and <scp><i>GLA</i></scp> gene sequencing
<scp><i>TBL1XR1</i></scp> associated intellectual disability, a new missense variant with dysmorphic features plus autism: Expanding the phenotypic spectrum
A new case of <scp>Smith‐Kingsmore</scp> syndrome with somatic <scp>MTOR</scp> pathogenic variant expands the phenotypic spectrum to lateralized overgrowth
Hematological findings associated with tubulin‐folding cofactors D‐related encephalopathy: Expanding the phenotype
<scp>Skraban‐Deardorff</scp> syndrome: Six new cases of <scp><i>WDR</i>26</scp>‐related disease and expansion of the clinical phenotype
Biallelic loss of function variant in the unfolded protein response gene <i>PDIA6</i> is associated with asphyxiating thoracic dystrophy and <scp>neonatal‐onset</scp> diabetes
Two intronic cis‐acting variants in both alleles of the <scp><i>POLR3A</i></scp> gene cause progressive spastic ataxia with hypodontia
Further delineation of the <scp><i>NTHL1</i></scp> associated syndrome: A report from the French Oncogenetic Consortium
Mutation in <scp><i>PHACTR1</i></scp> associated with multifocal epilepsy with infantile spasms and hypsarrhythmia
The sodium/proton exchanger <scp>SLC9C1</scp> (<scp>sNHE</scp>) is essential for human sperm motility and fertility
Bi‐allelic missense variant, p.<scp>Ser35Leu</scp> in <scp><i>EXOSC1</i></scp> is associated with pontocerebellar hypoplasia
Fragile X premutation and associated health conditions: A review
Expanded phenotypic spectrum of <scp><i>JAG1</i></scp>‐associated diseases: Central conducting lymphatic anomaly with a pathogenic variant in <scp><i>JAG1</i></scp>
Novel mutations in <scp><i>ZP1</i></scp>: Expanding the mutational spectrum associated with empty follicle syndrome in infertile women
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Adding evidence to the role of <scp><i>NEUROG1</i></scp> in congenital cranial dysinnervation disorders
Early genetic screening uncovered a high prevalence of thalassemia among 18 309 neonates in Guizhou, China
Achalasia as a symptom guide in MIRAGE syndrome: A novel case with p.R1293Q and p.R902W variants in the <i>SAMD9</i> gene
Clinical and neuroimaging findings in 33 patients with <scp>MCAP</scp> syndrome: A survey to evaluate relevant endpoints for future clinical trials
A Brazilian case arising from a homozygous canonical splice site <scp><i>SLC35A3</i></scp> variant leading to an in‐frame deletion
Congenital cervical spine malformation due to bi‐allelic <scp>RIPPLY2</scp> variants in spondylocostal dysostosis type 6
<scp>PLACK</scp> syndrome is potentially treatable with intralipids
Biallelic deletion in a minimal <scp><i>CAPN15</i></scp> intron in siblings with a recognizable syndrome of congenital malformations and developmental delay