Clinical Genetics - 2022

181 articles | Last updated: 2025-12-03 14:12:56
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Fatal congenital copper transport defect caused by a homozygous likely pathogenic variant of <scp><i>SLC31A1</i></scp>
<scp>ROSAH</scp> syndrome mimicking chronic uveitis
Novo pathogenic variations of <i>NLRP7</i> increasing the risk of gestational trophoblastic neoplasia
Chronic progressive external ophthalmoplegia plus syndrome due to homozygous missense variant in <i>TOP3A</i> gene
A novel biallelic nonsense variant in <scp><i>SLC38A3</i></scp> causing epileptic encephalopathy in an Indian family Indian family
Strategies for activity analysis of single nucleotide polymorphisms associated with human diseases
Genetic and clinical features of neonatal and early onset diabetes mellitus in a tertiary center cohort in Brazil
Natural history of clinical features in two brothers with acromesomelic dysplasia related to <i>PRKG2</i>
Novel homozygous variant in <scp><i>ARL2BP</i></scp> associated with retinitis pigmentosa, situs inversus, and male infertility in a Chinese patient Chinese
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A comprehensive functional analysis on the pathogenesis of novel <scp><i>TSPAN12</i></scp> and <scp><i>NDP</i></scp> variants in familial exudative vitreoretinopathy
Perinatal presentations of non‐immune hydrops fetalis due to recessive <i>PIEZO1</i> disease: A challenging fetal diagnosis
Novel de novo <scp>ZNF148</scp> truncating variant causing autism spectrum disorder, attention deficit hyperactivity disorder, and intellectual disability
A new case of Kaufman Oculocerebrofacial syndrome caused by two splicing variants in <scp><i>UBE3B</i></scp> and review of the literature
Identification of nonfunctional <scp>SPATA20</scp> causing acephalic spermatozoa syndrome in humans
Aminoacylation‐defective bi‐allelic mutations in human <scp>EPRS1</scp> associated with psychomotor developmental delay, epilepsy, and deafness
Putative founder effect of Arg338* <scp><i>AP4M1</i></scp> (<scp>SPG50</scp>) variant causing severe intellectual disability, epilepsy and spastic paraplegia: Report of three families Middle East; Turkish origin
Loss‐of‐function mutations in <scp><i>CST6</i></scp> cause dry skin, desquamation and abnormal keratosis without hypotrichosis Chinese origin
New insights in efficacy of different enzyme replacement therapy dosages in Fabry disease: Switch studies data following agalsidase beta shortage
Genetic screening in patients with ovarian dysfunction
Novel <scp><i>CNNM4</i></scp> variant and clinical features of <scp>J</scp>alili syndrome
Recurrent missense variants in clonal hematopoiesis‐related genes present in the general population
Bridging clinical care and research in Ontario, Canada: Maximizing diagnoses from reanalysis of clinical exome sequencing data
<i>FGF9</i> variant in 46,<scp>XY DSD</scp> patient suggests a role for dimerization in sex determination
A bi‐allelic missense change c.<scp>638A</scp> &gt; G in matrix metalloproteinase 15 in a patient with progressive familial intrahepatic cholestasis without cardiac anomalies
<scp><i>PIK3CA</i></scp> somatic mutations as potential biomarker for immunotherapy in elder or <scp><i>TP53</i></scp> mutated gastric cancer patients
A novel <scp><i>MAP3K7</i></scp> mutation in a child with cardiospondylocarpofacial syndrome and orofacial clefting
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Improving variant prioritization in exome analysis by entropy‐weighted ensemble of multiple tools
Dynamic interplay of <scp>microRNA</scp> in diseases and therapeutic
The identification of a novel <scp><i>CCNQ</i></scp> gene tail extension variant contributing to syndactyly, telecanthus and anogenital and renal malformations syndrome
Biallelic variants in <scp><i>CEP164</i></scp> cause a motile ciliopathy‐like syndrome
Management of copy number variants associated with incomplete penetrance and variable expressivity—Results of a French survey
A novel variant in <scp>AFF3</scp> underlying isolated syndactyly
Immunotherapy responsive neuroinflammation in a child with FAS‐associated death‐domain mutation
Expanding allelic and phenotypic spectrum of ZC4H2‐related disorder: A novel hypomorphic variant and high prevalence of tethered cord
New Cerebellar Ataxia, Neuropathy, Vestibular Areflexia Syndrome cases are caused by the presence of a nonsense variant in compound heterozygosity with the pathogenic repeat expansion in the <scp><i>R
De novo variants and recombination at 4q35: Hints for preimplantation genetic testing in facioscapulohumeral muscular dystrophy
Delineation of the clinical profile of <scp><i>CNOT2</i></scp> haploinsufficiency and overview of the <scp>IDNADFS</scp> phenotype
A novel biallelic variant c.<scp>2219T</scp> &gt; A p.(Leu740*) in <scp><i>ADGRG6</i></scp> as a cause of lethal congenital contracture syndrome 9
Vascular aneurysms in Ehlers‐Danlos syndrome subtypes: A systematic review
A systematic review on Treacher Collins syndrome: Correlation between molecular genetic findings and clinical severity Asian patients
Identification of variants in 94 Chinese patients with hereditary spherocytosis by next‐generation sequencing Chinese; Eastern China
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Six case reports of <scp><i>NTHL1</i></scp>‐associated tumor syndrome further support it as a multi‐tumor predisposition syndrome
Primary hyperoxaluria: Comprehensive mutation screening of the disease causing genes and spectrum of disease‐associated pathogenic variants Pakistani pediatric patients from South-Asian region
The constitutional gain‐of‐function variant p.<scp>Glu1099Lys</scp> in <i>NSD2</i> is associated with a novel syndrome
Biallelic variants in the <scp><i>SLC13A1</i></scp> sulfate transporter gene cause hyposulfatemia with a mild spondylo‐epi‐metaphyseal dysplasia
Copy‐number analysis by base‐level normalization: An intuitive visualization tool for evaluating copy number variations
A novel biallelic variant in the Popeye domain‐containing protein 1 (<scp>POPDC1</scp>) underlies limb girdle muscle dystrophy type 25
Loss‐of‐function mutations in <scp><i>SGCE</i></scp> found in Japanese patients with myoclonus‐dystonia Japanese
A novel variant in <scp><i>GATM</i></scp> causes idiopathic renal Fanconi syndrome and predicts progression to end‐stage kidney disease
Novel rare mutation in a conserved site of <scp><i>PTPRB</i></scp> causes human hypoplastic left heart syndrome
Cerebrotendinous xanthomatosis: A candidate for <scp>ACMG</scp> list of secondary findings?
Risk of genetic and epigenetic alteration in children conceived following <scp>ART</scp>: Is it time to return to nature whenever possible?
Fanconi anemia caused by biallelic inactivation of <scp><i>BRCA2</i></scp> can present with an atypical cancer phenotype in adulthood
A systematic review of the monogenic causes of Non‐Syndromic Hearing Loss (NSHL) and discussion of Current Diagnosis and Treatment options
Bi‐allelic pathogenic variants in <scp><i>ITGA8</i></scp> cause slowly progressive renal disease of unknown etiology
Platelet findings in 22q11.2 deletion syndrome correlate with disease manifestations but do not correlate with <scp>GPIb</scp> surface expression
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Splice‐site variant in the <scp><i>RPS7</i></scp> 5′‐<scp>UTR</scp> leads to a decrease in the <scp>mRNA</scp> level and development of <scp>Diamond‐Blackfan</scp> anemia
Six new cases of <scp>CRB2‐related</scp> syndrome and a review of clinical findings in 28 reported patients
Genetic overview of postaxial polydactyly: Updated classification
Expanding the spectrum of <i>KCNJ6</i>‐related disorders: Milder phenotype with pathological startle responses
Literature review on genotype–phenotype correlation in patients with hereditary spherocytosis Asian countries
Expanding the genetic spectrum of tooth agenesis using whole‐exome sequencing Chinese
A cross‐sectional study on fatigue, anxiety, and symptoms of depression and their relation with medical status in adult patients with <scp>M</scp>arfan syndrome. Psychological consequences in <scp>M</ general Dutch population
Novel stop‐gain <scp><i>RNF170</i></scp> variation detected in a Chinese family with adolescent‐onset hereditary spastic paraplegia Chinese
Hereditary spastic paraparesis presenting as cerebral palsy due to <scp><i>ADD3</i></scp> variant with mechanistic insight provided by a <i>Drosophila</i> γ‐adducin model
The oligogenic model of amyotrophic lateral sclerosis; phenotypes of three Tunisian families
Dysferlinopathies: Clinical and genetic variability
<scp>Diamond‐Blackfan</scp> anaemia caused by a <i>de novo</i> initiation codon mutation resulting in a shorter isoform of GATA1 East Asian population
A common intronic single nucleotide variant modifies <scp><i>PKD1</i></scp> expression level
Biallelic frameshift variant in the <scp><i>TBC1D2B</i></scp> gene in two siblings with progressive gingival overgrowth, fibrous dysplasia of face, and mental deterioration
A Gardos channelopathy associated with nonimmune hydrops and fetal loss
Psychiatric genetic counseling for people with copy number variants associated with psychiatric conditions
Common and rare variants in patients with early onset drusen maculopathy
Variants in <i>HNRNPH1</i> are associated with high myopia in humans and ocular coloboma in zebrafish
Consanguineous families with unusually high recurrence risk: A voice to be heard in the germline gene‐editing debate
A recurrent homozygous missense <scp><i>DPM3</i></scp> variant leads to muscle and brain disease
Looking closely at overgrowth: Constitutional mosaicism in <i>PTEN</i> hamartoma tumor syndrome
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Novel <i>PPP1R13L</i> variant expands the phenotype of a rare cardiocutaneous syndrome
Novel mutation in <scp><i>TTC21A</i></scp> triggers partial nonsense‐mediated <scp>mRNA</scp> decay and causes male infertility with <scp>MMAF</scp>
The recurrent <scp><i>TCF4</i></scp> missense variant p.(<scp>Arg389Cys</scp>) causes a neurodevelopmental disorder overlapping with but not typical for <scp>Pitt‐Hopkins</scp> syndrome
<scp>HIDEA</scp> syndrome is caused by biallelic, pathogenic, rare or founder <i>P4HTM</i> variants impacting the active site or the overall stability of the <scp>P4H‐TM</scp> protein Finnish ("Finnish enriched pathogenic P4HTM variants")
Microduplication of <scp><i>BTRC</i></scp> detected in a Chinese family with split hand/foot malformation type 3
Deep intronic variant in <i>MVK</i> as a cause for mevalonic aciduria initially presenting as non‐syndromic retinitis pigmentosa
<scp>HINT1</scp> neuropathy: Expanding the genotype and phenotype spectrum French
Identification of novel rare copy number variants associated with sporadic tetralogy of Fallot and clinical implications Han Chinese
Nomograms for prognostic risk assessment in glioblastoma multiforme: Applications and limitations
<scp><i>PSMC1</i></scp> variant causes a novel neurological syndrome
Expanding the phenotype of <i><scp>DNAJC30</scp>‐</i>associated Leigh syndrome
De novo 4q35.2 duplication containing <scp><i>FAT1</i></scp> is associated with autism spectrum disorder
A de novo inframe deletion variant in <scp><i>CAPZA2</i></scp> tentacle domain with global developmental delay and secondary microcephaly
A novel splice site variant in the <scp><i>POPDC3</i></scp> causes autosomal recessive limb‐girdle muscular dystrophy type 26
Clinical and molecular characterization of patients affected by <scp>Beckwith‐Wiedemann</scp> spectrum conceived through assisted reproduction techniques
Evolutionary origin of pathogenic <scp>GJB2</scp> alleles in China
A biallelic loss‐of‐function <i>PDIA6</i> variant in a second patient with polycystic kidney disease, infancy‐onset diabetes, and microcephaly
Neurocognitive and neurobehavioral characterization of two frequent forms of neurodevelopmental disorders: the <scp>DYRK1A</scp> and the <scp>Wiedemann–Steiner</scp> syndromes
Toxic and nutritional factors trigger Leber hereditary optic neuropathy due to a mitochondrial tRNA mutation
Genetics of <scp>non‐isolated</scp> hemivertebra: A systematic review of fetal, neonatal, and infant cases
A new <scp><i>SMOC2</i></scp> mutation within selective tooth agenesis, malformed teeth and dentin dysplasia
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Neuronal ceroïd‐lipofuscinosis: Clinical, electroencephalographic, imaging, and genetic study of a maghrebian series maghrebian
Expanding the clinical spectrum of primary coenzyme Q10 deficiency type 6: The first case with cardiomyopathy
Establishing the relationship between familial dysbetalipoproteinemia and genetic variants in the<scp><i>APOE</i></scp>gene
Functional characterization of a <scp><i>JAG1</i></scp> 5'<scp>UTR</scp> variant in a patient with clinically observed Alagille syndrome
Mutations in <scp><i>LRP5</i></scp> and <scp><i>BMP4</i></scp> are associated with mesiodens, tooth agenesis, root malformation, and oral exostoses
The first Chinese case of Siddiqi syndrome caused by a homozygous <i>FITM2</i> variant
Czech family confirms the new 1p36.13‐1p36.12 microdeletion syndrome
Clinical and genetic characterization of <scp><i>CACNA1A</i></scp>‐related disease
Disorders of histone methylation: Molecular basis and clinical syndromes
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Clinical and molecular genetic findings of Crisponi/cold‐induced sweating syndrome (CS/CISS) spectrum in patients from Turkey
Carriers of <scp><i>COL3A1</i></scp> pathogenic variants in Denmark: Interfamilial variability in severity and outcome of elective surgical procedures
Novel <scp><i>BMP1</i></scp>, <scp><i>CRTAP</i></scp>, and <scp><i>SERPINF1</i></scp> variants causing autosomal recessive osteogenesis imperfecta
Prenatal diagnosis of a germline variant in <scp><i>TRAF7</i></scp>: Importance of accessibility to prenatal exome sequencing in cases of structural fetal anomalies
Progressive frontal intraosseous lipoma: Detection of the mosaic <scp><i>AKT1</i></scp> variant discloses <scp>Proteus</scp> syndrome
Further characterization of <scp>Borjeson‐Forssman‐Lehmann</scp> syndrome in females due to de novo variants in <scp><i>PHF6</i></scp>
Estimation of familial recurrence risk of malignancy: Application of liability threshold model in genetic counseling
Further evidence for distinct traits associated with <scp>RBM10</scp> missense variants
Biallelic variants in <scp><i>ZNF142</i></scp> lead to a syndromic neurodevelopmental disorder
Further validation of the Perceptions of Uncertainties in Genome Sequencing scale among patients with cancer undergoing tumor sequencing
Biallelic variants in <scp><i>PAX3</i></scp> cause Klein syndrome
Novel <scp><i>LNPK</i></scp> variant causes progressive cerebral atrophy: Expanding the clinical phenotype
Molecular diagnosis of adult patients with clinically unexplained hypokalemia without hypertension demonstrated a diagnostic yield of 30.5%
A humanized murine model, demonstrating dominant progressive hearing loss caused by a novel <scp>KCNQ4</scp> mutation (p.<scp>G228D</scp>) from a large Chinese family Chinese
Expanding the novel <scp><i>MAPKAPK5</i></scp>–related developmental disorder's genotype–phenotype correlation: Patient report and 19 months of follow‐up
Loss of function mutation in <i>DNAH7</i> induces male infertility associated with abnormalities of the sperm flagella and mitochondria in human
Decipher non‐canonical <scp><i>SPAST</i></scp> splicing mutations with the help of functional assays in patients affected by spastic paraplegia 4 (<scp>SPG4</scp>)
Carrier frequency of autosomal recessive genetic conditions in diverse populations: Lessons learned from the genome aggregation database Amish, Finnish, Middle Eastern, diverse populations
Phenotypic expansion and variable expressivity in individuals with <i>JARID2</i>‐related intellectual disability: A case series
Genetic clinicians' confidence in <scp>BOADICEA</scp> comprehensive breast cancer risk estimates and counselees' psychosocial outcomes: A prospective study
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Understanding the new <scp><i>BRD4</i></scp>‐related syndrome: Clinical and genomic delineation with an international cohort study
A recurrent <i>ZP1</i> variant is responsible for oocyte maturation defect with degenerated oocytes in infertile females North African
A syndrome of severe intellectual disability, hypotonia, failure to thrive, dysmorphism, and thinning of corpus callosum maps to chromosome 7q21.13‐q21.3
<scp>SHP2</scp>'s gain‐of‐function in <scp>Werner</scp> syndrome causes childhood disease onset likely resulting from negative genetic interaction
A novel <scp>c.952T&gt;C</scp> mutation in <i>Notch3</i> gene in a patient with chronic non‐migraine‐like headache: Expanding the genotypic spectrum of <scp>CADASIL</scp>?
The clinical and biochemical hallmarks generally associated with <scp>GLUT1DS</scp> may be caused by defects in genes other than <scp><i>SLC2A1</i></scp>
<scp>CCDC82</scp> frameshift mutation associated with intellectual disability, spastic paraparesis, and dysmorphic features
<scp>IQSEC2</scp>‐related encephalopathy in males due to missense variants in the pleckstrin homology domain
Spinal muscular atrophy with congenital bone fractures 2 caused by a rare loss‐of‐function <i>ASCC1</i> gene mutation in two Bulgarian Roma patients Bulgarian Roma
Expanding genotypic and phenotypic spectrums of <scp><i>LTBP3</i></scp> variants in dental anomalies and short stature syndrome
<scp>El‐Hattab‐Alkuraya</scp> syndrome caused by biallelic <scp><i>WDR45B</i></scp> pathogenic variants: Further delineation of the phenotype and genotype
De novo heterozygous variants in <i>KIF5B</i> cause kyphomelic dysplasia
Pathogenic variant‐based preconception carrier screening in the <scp>Israeli Jewish</scp> population Israeli Jewish population; Jewish ethnicities; Arab, Druze and Bedouin populations; self-reported an
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Bi‐allelic <scp><i>SPATA22</i></scp> variants cause premature ovarian insufficiency and nonobstructive azoospermia due to meiotic arrest
A null founder variant in <scp><i>NPNT</i></scp>, encoding nephronectin, causes autosomal recessive renal agenesis
A novel homozygous variant in <scp><i>COX5A</i></scp> causes an attenuated phenotype with failure to thrive, lactic acidosis, hypoglycemia, and short stature
A homozygous frame‐shift variant in <scp><i>PROSER1</i></scp> is associated with developmental delay, hypotonia, genitourinary malformations, and distinctive facial features
Phenotypic spectrum of <i><scp>BLM</scp>‐</i> and <scp><i>RMI1</i></scp>‐related Bloom syndrome
The diagnosis of the first‐documented intragenic <scp><i>KANSL1</i></scp> microduplication patient broadens the genetic spectrum of Koolen de Vries syndrome
Neurofibromas in <scp><i>LZTR1</i></scp> schwannomatosis
<i>In Vitro</i> Fertilization assisted by IntraCytoplasmic Sperm Injection in a male patient with Bardet–Biedl syndrome
First evidence of <scp><i>SOX2</i></scp> mutations in Peters' anomaly: Lessons from molecular screening of 95 patients
Vertebral segmentation defects in a Brazilian cohort: Clinical and molecular analysis focused on spondylocostal dysostosis
Systemic inflammatory syndrome in children with <scp><i>FARSA</i></scp> deficiency
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Kidney failure in <scp>Bardet</scp>–<scp>Biedl</scp> syndrome
Mitochondrial tRNA<sup>His</sup>mutation (m.12158A &gt; G) associated with MELAS syndrome
Functional loss of ubiquitin‐specific protease 14 may lead to a novel distal arthrogryposis phenotype
Multicenter study on the genetics of glomerular diseases among southeast and south Asians: Deciphering Diversities ‐ Renal Asian Genetics Network (DRAGoN)
<scp>CIBERER</scp>: Spanish national network for research on rare diseases: A highly productive collaborative initiative
De novo variants in <scp><i>AGO1</i></scp> recapitulate a heterogeneous neurodevelopmental disorder phenotype
Late‐onset hearing loss case associated with a heterozygous truncating variant of <scp><i>DIAPH1</i></scp>
An adult male with <scp><i>SHANK2</i></scp> variant with epilepsy and obsessive‐compulsive disorder: Expanding the shankopathy phenotypic spectrum
Prevalence of bladder cancer in Costello syndrome: New insights to drive clinical decision‐making
Genetic risk of hyperuricemia in hypertensive patients associated with antihypertensive drug therapy: A longitudinal study Chinese
<scp>Bi‐allelic</scp> null variant in matrix metalloproteinase‐15, causes congenital cardiac defect, cholestasis jaundice, and failure to thrive
A new phenotype of syndromic retinitis pigmentosa with myopathy is caused by mutations in retinol dehydrogenase 11 Chinese patient; Chinese population
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Corrigendum
Di‐genic inheritance of germline <i>POLE</i> and <i>PMS2</i> pathogenic variants causes a unique condition associated with pediatric cancer predisposition
A splice site mutation in the <scp><i>TSEN2</i></scp> causes a new syndrome with craniofacial and central nervous system malformations, and atypical hemolytic uremic syndrome
Pathogenic “germline” variants associated with myeloproliferative disorders in apparently normal individuals: Inherited or acquired genetic alterations?
Divergent variant patterns among 19 patients with<scp>Rubinstein‐Taybi</scp>syndrome uncovered by comprehensive genetic analysis including whole genome sequencing
The risks of breast and ovarian cancer associated with the Ashkenazi Jewish founder allele <scp><i>BRCA2</i> 6174delT</scp> Ashkenazi Jewish; non-Jewish