Human Mutation - 2021

162 articles | Last updated: 2025-12-03 14:12:57
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Mutations in <i>KIF7</i> implicated in idiopathic scoliosis in humans and axial curvatures in zebrafish
GeneBreaker: Variant simulation to improve the diagnosis of Mendelian rare genetic diseases
Functional and clinical implications of genetic structure in 1686 Italian exomes
Expanding the clinical and genetic spectrum of FDXR deficiency by functional validation of variants of uncertain significance
Issue Information
Functionally deficient <i>TRPV6</i> variants contribute to hereditary and familial chronic pancreatitis
<i>EFEMP1</i> rare variants cause familial juvenile‐onset open‐angle glaucoma
Vulnerable exons, like <i>ACADM</i> exon 5, are highly dependent on maintaining a correct balance between splicing enhancers and silencers
Novel biallelic mutations in<i>SLC26A8</i>cause severe asthenozoospermia in humans owing to midpiece defects: Insights into a putative dominant genetic disease
<i>ROHMM</i>—A flexible hidden Markov model framework to detect runs of homozygosity from genotyping data
Harmonizing variant classification for return of results in the All of Us Research Program
A decade of <i>RAD51C</i> and <i>RAD51D</i> germline variants in cancer
Generation and mutational analysis of a transgenic mouse model of human<i>SRY</i>
Response to: Phenotypic heterogeneity of Leigh syndrome due to <i>NDUFA12</i> variants is multicausal
Phenotypic heterogeneity of Leigh syndrome due to <i>NDUFA12</i> variants is multicausal
Unraveling the molecular basis underlying nine putative splice site variants of von Willebrand factor
Variant calling: Considerations, practices, and developments
Long‐read whole genome sequencing reveals HOXD13 alterations in synpolydactyly
The enhancer rare germline variation rs548071605 contributes to lung cancer development
Variant interpretation using population databases: Lessons from gnomAD
The clinical and molecular spectrum of <i>QRICH1</i> associated neurodevelopmental disorder
Recommendations by the ClinGen Rett/Angelman‐like expert panel for gene‐specific variant interpretation methods
Imaging‐based evaluation of pathogenicity by novel <i>DNM2</i> variants associated with centronuclear myopathy
<i>NR2F1</i>database: 112 variants and 84 patients support refining the clinical synopsis of Bosch–Boonstra–Schaaf optic atrophy syndrome
Pseudoexon activation in disease by non‐splice site deep intronic sequence variation — wild type pseudoexons constitute high‐risk sites in the human genome
Annotating and prioritizing genomic variants using the Ensembl Variant Effect Predictor—A tutorial
GGCX variants leading to biallelic deficiency to γ‐carboxylate GRP cause skin laxity in VKCFD1 patients
A novel gene mutation in <i>ZP3</i> loop region identified in patients with empty follicle syndrome
Constitutional <i>POLE</i> variants causing a phenotype reminiscent of constitutional mismatch repair deficiency
Novel PHEX gene locus‐specific database: Comprehensive characterization of vast number of variants associated with X‐linked hypophosphatemia (XLH)
Issue Information
Cover, Volume 42, Issue 12
Identification of novel deep intronic <i>PAH</i> gene variants in patients diagnosed with phenylketonuria
Clinical variability at the mild end of <i>BRAT1</i> ‐related spectrum: Evidence from two families with genotype–phenotype discordance
Targeted massively parallel sequencing of candidate regions on chromosome 22q predisposing to multiple schwannomas: An analysis of 51 individuals in a single‐center experience
Utilizing ClinGen gene‐disease validity and dosage sensitivity curations to inform variant classification
Analysis of patient‐specific <i>NF1</i> variants leads to functional insights for Ras signaling that can impact personalized medicine
Issue Information
Cover, Volume 42, Issue 11
A family study implicates <i>GBE1</i> in the etiology of autism spectrum disorder
Duplications in the G3 domain or switch II region in <i>HRAS</i> identified in patients with Costello syndrome
A comprehensive molecular study identified 12 complementation groups with 56 novel FANC gene variants in Indian Fanconi anemia subjects
A bi‐allelic loss‐of‐function <i>SARS1</i> variant in children with neurodevelopmental delay, deafness, cardiomyopathy, and decompensation during fever
Correlation between<i>FBN1</i>mutations and ocular features with ectopia lentis in the setting of Marfan syndrome and related fibrillinopathies
Molecular landscape of<i>DYSF</i>mutations in dysferlinopathy: From a Chinese multicenter analysis to a worldwide perspective
Adapting the ACMG/AMP variant classification framework: A perspective from the ClinGen Hemoglobinopathy Variant Curation Expert Panel
Noncoding sequence variants define a novel regulatory element in the first intron of the <i>N</i> ‐acetylglutamate synthase gene
Epidemiological aspects of hereditary fructose intolerance: A database study
Issue Information
Validation of low‐coverage whole‐genome sequencing for mitochondrial DNA variants suggests mitochondrial DNA as a genetic cause of preterm birth
Actionable genomic variants in 6045 participants from the Qatar Genome Program
VIP‐HL: Semi‐automated ACMG/AMP variant interpretation platform for genetic hearing loss
Issue Information
Altered regulation of <i>BRCA1</i> exon 11 splicing is associated with breast cancer risk in carriers of <i>BRCA1</i> pathogenic variants
<i>PRPH2</i> mutation update: In silico assessment of 245 reported and 7 novel variants in patients with retinal disease
Identification of incompletely penetrant variants and interallelic interactions in autosomal recessive disorders by a population‐genetic approach
Genome sequencing and RNA‐seq analyses of mitochondrial complex I deficiency revealed <i>Alu</i> insertion‐mediated deletion in <i>NDUFV2</i>
Broad variation in phenotypes for common <i>GAA</i> genotypes in Pompe disease
Molecular basis of mucopolysaccharidosis IVA (Morquio A syndrome): A review and classification of <i>GALNS</i> gene variants and reporting of 68 novel variants
GATA2 deficiency syndrome: A decade of discovery
A domain damage index to prioritizing the pathogenicity of missense variants
<i>Fumarate Hydratase</i> ( <i>FH</i> ) c.1431_1433dupAAA (p.Lys477dup) variant is not associated with FH protein deficiency and increased 2SC in two separate patie
Expanding the genotypes and phenotypes for 19 rare diseases by exome sequencing performed in pediatric intensive care unit
SLC25A38 congenital sideroblastic anemia: Phenotypes and genotypes of 31 individuals from 24 families, including 11 novel mutations, and a review of the literature
Epistatic interaction of <i>PDE4DIP</i> and <i>DES</i> mutations in familial atrial fibrillation with slow conduction
Corrigendum
Issue Information
An updated quantitative model to classify missense variants in the <i>TP53</i> gene: A novel multifactorial strategy
Pathogenic noncoding variants in the neurofibromatosis and schwannomatosis predisposition genes
Lack of <i>MECP2</i> gene transcription on the duplicated alleles of two related asymptomatic females with Xq28 duplications and opposite X‐chromosome inactivation skewing
Functional characterization of novel variants in <i>SMPD1</i> in Indian patients with acid sphingomyelinase deficiency
Clinical and molecular characteristics of 168 probands and 65 relatives with a clinical presentation of classical Ehlers–Danlos syndrome
Variants of human <i>CLDN9</i> cause mild to profound hearing loss
YMrCA: Improving Y‐chromosomal ancestor time estimation for DNA kinship research
The prevalence of HLA‐I LOH in Chinese pan‐cancer patients and genomic features of patients harboring HLA‐I LOH
A custom capture sequence approach for oculocutaneous albinism identifies structural variant alleles at the <i>OCA2</i> locus
Case–case analysis addressing ascertainment bias for multigene panel testing implicates <i>BRCA1</i> and <i>PALB2</i> in endometrial cancer
Genetic landscape of recessive diseases in the Vietnamese population from large‐scale clinical exome sequencing
A discarded synonymous variant in <i>NPHP3</i> explains nephronophthisis and congenital hepatic fibrosis in several families
Muscarinic acetylcholine receptor M1 mutations causing neurodevelopmental disorder and epilepsy
Elucidating clinical phenotypic variability associated with the polyT tract and TG repeats in CFTR
Issue Information
Upstream ORF frameshift variants in the<i>PAX6</i>5ʹUTR cause congenital aniridia
CanVaS: Documenting the genetic variation spectrum of Greek cancer patients
Myopathy can be a key phenotype of membrin (GOSR2) deficiency
Genes affecting ionizing radiation survival identified through combined exome sequencing and functional screening
Global spectrum of population‐specific common missense variation in cytochrome P450 pharmacogenes
Identification of seven exonic variants in the <i>SLC4A1</i> , <i>ATP6V1B1</i> , and <i>ATP6V0A4</i> genes that alter RNA splici
Prevalence and phenotype associations of complement factor I mutations in geographic atrophy
A Functional Variant on 9p21.3 Related to Glioma Risk Affects Enhancer Activity and Modulates Expression of <i>CDKN2B‐AS1</i>
Further evidence for <i>de novo</i> variants in <i>SYNCRIP</i> as the cause of a neurodevelopmental disorder
Genome sequencing in congenital cataracts improves diagnostic yield
Novel <i>ALDH3A2</i> mutations in structural and functional domains of FALDH causing diverse clinical phenotypes in Sjögren–Larsson syndrome patients
Biallelic variants in <i>RNU12</i> cause CDAGS syndrome
Common and rare myocilin variants: Predicting glaucoma pathogenicity based on genetics, clinical, and laboratory misfolding data
Integrated in silico and experimental assessment of disease relevance of <i>PCDH19</i>  missense variants
Biallelic deep intronic variant c.5457+81T&gt;A in <i>TRIP11</i> causes loss of function and results in achondrogenesis 1A
Incorporation of exome‐based CNV analysis makes trio‐WES a more powerful tool for clinical diagnosis in neurodevelopmental disorders: A retrospective study
Common polymorphic <i>OTC</i> variants can act as genetic modifiers of enzymatic activity
Dissection of contiguous gene effects for deletions around <i>ERF</i> on chromosome 19
Issue Information
Genetic pleiotropy of <i>ERCC6</i> loss‐of‐function and deleterious missense variants links retinal dystrophy, arrhythmia, and immunodeficiency in diverse ancestries diverse ancestries
Extending the allelic spectrum at noncoding risk loci of orofacial clefting
A novel <i>GSN</i> variant outside the G2 calcium‐binding domain associated with Amyloidosis of the Finnish type Finnish
New insights into the role of endoplasmic reticulum‐associated degradation in Bartter Syndrome Type 1
Identification of missense <i>MAB21L1</i> variants in microphthalmia and aniridia
Exome sequencing as the first‐tier test for pediatric respiratory diseases: A single‐center study
Bi‐allelic <i>KARS1</i> pathogenic variants affecting functions of cytosolic and mitochondrial isoforms are associated with a progressive and multisystem disease
Benchmarking deep learning splice prediction tools using functional splice assays
Broadening the phenotypic spectrum and physiological insights related to<i>EIF2S3</i>variants
von Willebrand factor propeptide missense variants affect anterograde transport to Golgi resulting in ER retention
New cases that expand the genotypic and phenotypic spectrum of Congenital NAD Deficiency Disorder
Issue Information
Cover, Volume 42, Issue 5
Cover, Volume 42, Issue 4
Novel missense mutations in PTCHD1 alter its plasma membrane subcellular localization and cause intellectual disability and autism spectrum disorder
Issue Information
Clinical, neuroimaging, and molecular spectrum of <i>TECPR2</i> ‐associated hereditary sensory and autonomic neuropathy with intellectual disability
Different types of disease‐causing noncoding variants revealed by genomic and gene expression analyses in families with X‐linked intellectual disability
<i>CNGB1</i> ‐related rod‐cone dystrophy: A mutation review and update
Prediction of disease‐associated functional variants in noncoding regions through a comprehensive analysis by integrating datasets and features
Haploinsufficiency of <i>POU4F1</i> causes an ataxia syndrome with hypotonia and intention tremor
More than meets the eye: Expanding and reviewing the clinical and mutational spectrum of brittle cornea syndrome
Beyond copy number: A new, rapid, and versatile method for sequencing the entire <i>SMN2</i> gene in SMA patients
Lung‐specific distant enhancer cis regulates expression of<i>FOXF1</i>and lncRNA<i>FENDRR</i>
Novel <i>NDUFA12</i> variants are associated with isolated complex I defect and variable clinical manifestation
Implementation of multigene panel NGS diagnosis in the national primary ciliary dyskinesia cohort of Cyprus: An island with a high disease prevalence
KATK: Fast genotyping of rare variants directly from unmapped sequencing reads
Altered DNA repair creates novel Alu/Alu repeat‐mediated deletions
Comprehensive characterization of <i>Alu</i> ‐mediated breakpoints in germline <i>VHL</i> gene deletions and rearrangements in patients from 71 VHL fami
Clinical, biochemical, and genotype‐phenotype correlations of 118 patients with Niemann‐Pick disease Types A/B
A single NGS‐based assay covering the entire genomic sequence of the <i>DMD</i> gene facilitates diagnostic and newborn screening confirmatory testing
Phenotypic and protein localization heterogeneity associated with <i>AHDC1</i> pathogenic protein‐truncating alleles in Xia–Gibbs syndrome
The phenotypic and genetic spectrum of patients with heterozygous mutations in cyclin M2 (CNNM2)
Loss‐of‐function variants in <i>ARHGEF9</i> are associated with an X‐linked intellectual disability dominant disorder
Corrigendum
A global analysis of the reconstitution of PTEN function by translational readthrough of<i>PTEN</i>pathogenic premature termination codons
Genomic variants causing mitochondrial dysfunction are common in hereditary lower motor neuron disease
Spectrum of pathogenic variants and founder effects in amelogenesis imperfecta associated with <i>MMP20</i>
Considerations for using population frequency data in germline variant interpretation: Cancer syndrome genes as a model
Diversity of functional alterations of the ClC‐5 exchanger in the region of the proton glutamate in patients with Dent disease 1
Re‐evaluating the pathogenicity of the c.783+2T&gt;C <i>BAP1</i> germline variant
MutSpliceDB: A database of splice sites variants with RNA‐seq based evidence on effects on splicing
Biallelic hypomorphic variants in ALDH1A2 cause a novel lethal human multiple congenital anomaly syndrome encompassing diaphragmatic, pulmonary, and cardiovascular defects
TAOK1 is associated with neurodevelopmental disorder and essential for neuronal maturation and cortical development
Cancer SIGVAR: A semiautomated interpretation tool for germline variants of hereditary cancer‐related genes
Issue Information
Novel variants in critical domains of ATP8A2 and expansion of clinical spectrum
The reversion variant (p.Arg90Leu) at the evolutionarily adaptive p.Arg90 site in CELA3B predisposes to chronic pancreatitis
<i>CHM</i> mutation spectrum and disease: An update at the time of human therapeutic trials
Relationship of <i>DUX4</i> and target gene expression in FSHD myocytes
Diagnostic and clinical utility of next‐generation sequencing in children born with multiple congenital anomalies in the China neonatal genomes project
Severe congenital lactic acidosis and hypertrophic cardiomyopathy caused by an intronic variant in <i>NDUFB7</i>
A data set of variants derived from 1455 clinical and research exomes is efficient in variant prioritization for early‐onset monogenic disorders in Indians
Cover, Volume 42, Issue 2
Erratum
Issue Information
Cover, Volume 42, Issue 2
Molecular characterization of pathogenic <i>OTOA</i> gene conversions in hearing loss patients
SNPs associated with colorectal cancer at 15q13.3 affect risk enhancers that modulate <i>GREM1</i> gene expression
The genomic landscape of pediatric rheumatology disorders in the Middle East
Functional characterization of <i>ABCC8</i> variants of unknown significance based on bioinformatics predictions, splicing assays, and protein analyses: Benefits for the accura