Human Mutation - 2020

191 articles | Last updated: 2025-12-03 14:12:57
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T A T A T A T A
Cover, Volume 42, Issue 1
Issue Information
Rare coding variants in <i>MAPK7</i> predispose to adolescent idiopathic scoliosis
The corrected breakpoint sequence of the homozygous <i>SPINK1</i> deletion causing severe infantile isolated exocrine pancreatic insufficiency
Whole‐exome sequencing of non‐ <i>BRCA1/BRCA2</i> mutation carrier cases at high‐risk for hereditary breast/ovarian cancer
Phenotypic differences of mutation‐negative cases in Gitelman syndrome clinically diagnosed in adulthood
Epidemiological and ES cell‐based functional evaluation of <i>BRCA2</i> variants identified in families with breast cancer
A new case of TAR syndrome confirms the importance of noncoding variants in the etiopathogenesis of the disease
Update of the Pompe variant database for the prediction of clinical phenotypes: Novel disease‐associated variants, common sequence variants, and results from newborn screening
Expanding the phenotype of X‐linked SSR4–CDG: Connective tissue implications
Specifications of the ACMG/AMP variant interpretation guidelines for germline <i>TP53</i> variants
Identification of SRSF10 as a regulator of <i>SMN2</i> ISS‐N1
New clinical and molecular evidence linking mutations in <i>ARSG</i> to Usher syndrome type IV
Mitochondrial genome variant m.3250T&gt;C as a possible risk factor for mitochondrial cardiomyopathy
Biallelic <i>TMEM251</i> variants in patients with severe skeletal dysplasia and extreme short stature
<i>GRIN</i> database: A unified and manually curated repertoire of <i>GRIN</i> variants
Verifying nomenclature of DNA variants in submitted manuscripts: Guidance for journals
Detection and validation of novel mutations in<i>MERTK</i>in a simplex case of retinal degeneration using WGS and hiPSC–RPEs model
New variants and in silico analyses in GRK1 associated Oguchi disease
A high‐content drug screening strategy to identify protein level modulators for genetic diseases: A proof‐of‐principle in autosomal dominant leukodystrophy
Cover, Volume 41, Issue 12
Issue Information
Aberrant <i>COL11A1</i> splicing causes prelingual autosomal dominant nonsyndromic hearing loss in the DFNA37 locus
Novel homozygous truncating variants in <i>ZMYND15</i> causing severe oligozoospermia and their implications for male infertility
A novel variant in <i>COX16</i> causes cytochrome c oxidase deficiency, severe fatal neonatal lactic acidosis, encephalopathy, cardiomyopathy, and liver dysfunction
The p.Ser64Leu and p.Pro104Leu missense variants of PALB2 identified in familial pancreatic cancer patients compromise the DNA damage response
A functional variant on 20q13.33 related to glioma risk alters enhancer activity and modulates expression of multiple genes
Multiomic analysis elucidates Complex I deficiency caused by a deep intronic variant in <i>NDUFB10</i>
Efficient detection of copy‐number variations using exome data: Batch‐ and sex‐based analyses
Next‐generation sequencing in a series of 80 fetuses with complex cardiac malformations and/or heterotaxy
Loss of CBY1 results in a ciliopathy characterized by features of Joubert syndrome
Relationship between sodium channel function and clinical phenotype in SCN5A variants associated with Brugada syndrome
De novo variants in <i>CELF2</i> that disrupt the nuclear localization signal cause developmental and epileptic encephalopathy
A commentary on “A Vietnamese human genetic variation database”
Erratum
Single nucleotide polymorphisms in <i>CEL‐HYB1</i> increase risk for chronic pancreatitis through proteotoxic misfolding
Heterogeneous phenotypic expression of <i>C1QBP</i> variants is attributable to variable heteroplasmy of secondary mtDNA deletions and mtDNA copy number
Response to: “Heterogeneous phenotypic expression of <i>C1QBP</i> variants is attributable to variable heteroplasmy of secondary mtDNA deletions and mtDNA copy number”
Cover, Volume 41, Issue 11
Issue Information
<i>USF3</i>modulates osteoporosis risk by targeting<i>WNT16</i>,<i>RANKL</i>,<i>RUNX2</i>, and two GWAS lead SNPs rs2908007 and rs4531631
Comprehensive analysis and ACMG‐based classification of <i>CHEK2</i> variants in hereditary cancer patients
<i>SMN1</i> copy‐number and sequence variant analysis from next‐generation sequencing data
FREQMAX provides an alternative approach for determining high‐resolution allele frequency thresholds in carrier screening
Overcoming presynaptic effects of VAMP2 mutations with 4‐aminopyridine treatment
Cytogenetically visible inversions are formed by multiple molecular mechanisms
Rare hypomorphic human variation in the heptahelical domain of<i>SMO</i>contributes to holoprosencephaly phenotypes
Issue Information
Cover, Volume 41, Issue 10
Rapid exome sequencing and adjunct RNA studies confirm the pathogenicity of a novel homozygous <i>ASNS</i> splicing variant in a critically ill neonate
Rescue of common exon‐skipping mutations in cystic fibrosis with modified U1 snRNAs
EuroGEMS.org: Guide and links to online genetic and genomic educational resources, valuable for all levels
Novel <i>F8</i> and <i>F9</i> gene variants from the PedNet hemophilia registry classified according to ACMG/AMP guidelines
Quantification of DNA methylation independent of sodium bisulfite conversion using methylation‐sensitive restriction enzymes and digital PCR
Breakpoint junction analysis for complex genomic rearrangements with the caldera volcano‐like pattern
Phenotypic expansion in <i>KIF1A</i> ‐related dominant disorders: A description of novel variants and review of published cases
Functional analysis of<i>Sonic Hedgehog</i>variants associated with holoprosencephaly in humans using a CRISPR/Cas9 zebrafish model
Common atrium/atrioventricular canal defect and postaxial polydactyly: A mild clinical subtype of Ellis‐van Creveld syndrome caused by hypomorphic mutations in the <i>EVC</i> g
Comparative analysis of rare <i>EDAR</i> mutations and tooth agenesis pattern in <i>EDAR</i> ‐ and <i>EDA</i> ‐associated nonsyn
A comprehensive bioinformatic analysis of 126 patients with an inherited platelet disorder to identify both sequence and copy number genetic variants
Frequency spectrum of rare and clinically relevant markers in multiethnic Indian populations (ClinIndb): A resource for genomic medicine in India
Specifications of the ACMG/AMP standards and guidelines for mitochondrial DNA variant interpretation
Functional analysis and classification of homozygous and hypomorphic ABCA4 variants associated with Stargardt macular degeneration
Cardiomyopathy‐associated mutations in the RS domain affect nuclear localization of RBM20
Goldberg–Shprintzen syndrome is determined by the absence, or reduced expression levels, of KIFBP
Retracted: Identification of seven novel mutations in ABCD1 by a DHPLC‐based assay in Italian patients with X‐linked adrenoleukodystrophy Italian patients
Novel genotype–phenotype correlation of functionally characterized <i>LMX1A</i> variants linked to sensorineural hearing loss
Renal dysfunction, rod‐cone dystrophy, and sensorineural hearing loss caused by a mutation in <i>RRM2B</i>
Expansion of germline <i>RPS20</i> mutation phenotype to include Diamond–Blackfan anemia
Functional consequences of <i>SLC1A3</i> mutations associated with episodic ataxia 6
Familial Mediterranean fever: Penetrance of the p.[Met694Val];[Glu148Gln] and p.[Met694Val];[=] genotypes
Large‐scale comparative evaluation of user‐friendly tools for predicting variant‐induced alterations of splicing regulatory elements
Cover, Volume 41, Issue 8
Issue Information
Response to: A commentary on “A Vietnamese human genetic variation database”
Cover, Volume 41, Issue 8
Fitting a naturally scaled point system to the ACMG/AMP variant classification guidelines
A commentary on “A Vietnamese human genetic variation database”
Cover, Volume 41, Issue 8
Cover, Volume 41, Issue 8
Splicing impact of deep exonic missense variants in <i>CAPN3</i> explored systematically by minigene functional assay
Expansion of the phenotypic spectrum of de novo missense variants in kinesin family member 1A ( <i>KIF1A</i> )
De novo mutation and skewed X‐inactivation in girl with <i>BCAP31</i> ‐related syndrome
Homozygous mutations in <i>C1QBP</i> as cause of progressive external ophthalmoplegia (PEO) and mitochondrial myopathy with multiple mtDNA deletions
Clinical and laboratory interpretation of mitochondrial mRNA variants
Long‐term uninterrupted enzyme replacement therapy prevents liver disease in murine model of severe homocystinuria
Biallelic loss‐of‐function variants in <i>TBC1D2B</i> cause a neurodevelopmental disorder with seizures and gingival overgrowth
Variant effect on splicing regulatory elements, branchpoint usage, and pseudoexonization: Strategies to enhance bioinformatic prediction using hereditary cancer genes as exemplars
EpigenCentral: Portal for DNA methylation data analysis and classification in rare diseases
The expanding clinical phenotype of germline <i>ABL1</i> ‐associated congenital heart defects and skeletal malformations syndrome
Human genes differ by their UV sensitivity estimated through analysis of UV‐induced silent mutations in melanoma
Mandibular‐pelvic‐patellar syndrome is a novel<i>PITX1</i>‐related disorder due to alteration of PITX1 transactivation ability
General population ZBTB18 missense variants influence DNA binding and transcriptional regulation
Revisiting the complex architecture of ALS in Turkey: Expanding genotypes, shared phenotypes, molecular networks, and a public variant database
Expanding the genotypic and phenotypic spectrum of severe serine biosynthesis disorders
Identification and characterization of novel rapidly mutating Y‐chromosomal short tandem repeat markers
A single c.1715G&gt;C calpain 3 gene variant causes dominant calpainopathy with loss of calpain 3 expression and activity
Issue Information
Genetic architecture of inherited retinal degeneration in Germany: A large cohort study from a single diagnostic center over a 9‐year period
Genotype–phenotype associations in a large <i>PRPH2</i> ‐related retinopathy cohort
An assessment of the role of vinculin loss of function variants in inherited cardiomyopathy
First clinical and myopathological description of a myofibrillar myopathy with congenital onset and homozygous mutation in <i>FLNC</i>
Xdrop: Targeted sequencing of long DNA molecules from low input samples using droplet sorting
Clinical and molecular genetic characterization of two female patients harboring the Xq27.3q28 deletion with different ratios of X chromosome inactivation
Integrated multi‐omics data analyses for exploring the co‐occurring and mutually exclusive gene alteration events in colorectal cancer
Suggested application of HER2+ breast tumor phenotype for germline <i>TP53</i> variant classification within ACMG/AMP guidelines
<i>ASAH1</i> pathogenic variants associated with acid ceramidase deficiency: Farber disease and spinal muscular atrophy with progressive myoclonic epilepsy
Candidate genes for hereditary colorectal cancer: Mutational screening and systematic review
The expanding<i>LARS2</i>phenotypic spectrum: HLASA, Perrault syndrome with leukodystrophy, and mitochondrial myopathy
Hypoparathyroidism, deafness, and renal dysplasia syndrome: 20 Years after the identification of the first<i>GATA3</i>mutations
Mutation prevalence tables for hereditary cancer derived from multigene panel testing
Identification of pathogenic variants of<i>ERLEC1</i>in individuals with Class III malocclusion by exome sequencing
AutoPVS1: An automatic classification tool for PVS1 interpretation of null variants
Single‐fiber studies for assigning pathogenicity of eight mitochondrial DNA variants associated with mitochondrial diseases
The genetic profile of dysferlinopathy in a cohort of 209 cases: Genotype–phenotype relationship and a hotspot on the inner DysF domain
Issue Information
Constraint and conservation of paired‐type homeodomains predicts the clinical outcome of missense variants of uncertain significance
<i>TRPV6</i> variants confer susceptibility to chronic pancreatitis in the Chinese population
Clinical validity of expanded carrier screening: Evaluating the gene‐disease relationship in more than 200 conditions
5′ splice site GC&gt;GT and GT&gt;GC variants differ markedly in terms of their functionality and pathogenicity
An update on genetic variants of the<i>NKX2‐5</i>
Bi‐allelic mutations in <i>HARS1</i> severely impair histidyl‐tRNA synthetase expression and enzymatic activity causing a novel multisystem ataxic syndrome
The EAHAD blood coagulation factor VII variant database
Pathogenic variants of <i>DNAJC12</i> and evaluation of the encoded cochaperone as a genetic modifier of hyperphenylalaninemia
<i>EFTUD2</i> missense variants disrupt protein function and splicing in mandibulofacial dysostosis Guion‐Almeida type
Rare mutations in the autophagy‐regulating gene <i>AMBRA1</i> contribute to human neural tube defects
Issue Information
Concern regarding classification of c.703G&gt;A/p.Gly235Arg as a novel missense variant in <i>KRIT1</i> gene
Response to: Concern regarding classification of c.703G&gt;A/p.Gly235Arg as a novel missense variant in <i>KRIT1</i> gene
Front Cover, Volume 41, Issue 5
Documentation of clinically relevant genomic biomarker allele frequencies in the next‐generation FINDbase worldwide database
Null phenotype of neurofibromatosis type 1 in a carrier of a heterozygous atypical <i>NF1</i> deletion due to mosaicism
TAR syndrome: Clinical and molecular characterization of a cohort of 26 patients and description of novel noncoding variants of <i>RBM8A</i>
<i>CACNA1H</i> variants are not a cause of monogenic epilepsy
dbMTS: A comprehensive database of putative human microRNA target site SNVs and their functional predictions
Disease‐causing missense mutations within the N‐terminal transmembrane domain of GlcNAc‐1‐phosphotransferase impair endoplasmic reticulum translocation or Golgi retention
Characterization of intellectual disability and autism comorbidity through gene panel sequencing
Damaging de novo missense variants in<i>EEF1A2</i>lead to a developmental and degenerative epileptic‐dyskinetic encephalopathy
A protective polymorphism in<i>MMP16</i>, improved blood gas levels, and chronic obstructive pulmonary diseases: Family and two population‐based studies
Functional characterization of four ATP‐binding cassette transporter A3 gene (<i>ABCA3</i>) variants
Targeted resequencing reveals rare variants enrichment in multiple sclerosis susceptibility genes
LEAP: Using machine learning to support variant classification in a clinical setting
Issue Information
Large‐scale in vitro functional testing and novel variant scoring via protein modeling provide insights into alkaline phosphatase activity in hypophosphatasia
<i>Int22h1/Int22h2</i> ‐mediated Xq28 duplication syndrome: de novo duplications, prenatal diagnoses, and additional phenotypic features
A mutation update for the <i>FLNC</i> gene in myopathies and cardiomyopathies
TMEM16E/ANO5 mutations related to bone dysplasia or muscular dystrophy cause opposite effects on lipid scrambling
Pathogenic <i>PTPN11</i> variants involving the poly‐glutamine Gln <sup>255</sup> ‐Gln <sup>256</sup> ‐Gln <sup>257<
AG‐exclusion zone revisited: Lessons to learn from 91 intronic NF1 3′ splice site mutations outside the canonical AG‐dinucleotides
Type II Alexander disease caused by splicing errors and aberrant overexpression of an uncharacterized GFAP isoform
Missense variants in <i>TAF1</i> and developmental phenotypes: Challenges of determining pathogenicity
The X‐linked filaminopathies: Synergistic insights from clinical and molecular analysis
<i>ZMYND11</i> ‐related syndromic intellectual disability: 16 patients delineating and expanding the phenotypic spectrum
<i>Retraction</i> : Exploring the potential role of disease‐causing mutation in a gene desert: Duplication of noncoding elements 5′ of <i>GRIA3</i> is associated wi
Exome sequencing identifies the first genetic determinants of sirenomelia in humans
<i>FANCL</i> gene mutations in premature ovarian insufficiency
Critical assessment of secondary findings in genes linked to primary arrhythmia syndromes
Update of variants identified in the pancreatic β‐cell K <sub>ATP</sub> channel genes <i>KCNJ11</i> and <i>ABCC8</i> in individu
Issue Information
Front Cover, Volume 41, Issue 3
<i>BAZ2B</i> haploinsufficiency as a cause of developmental delay, intellectual disability, and autism spectrum disorder
Functional characterization of the first missense variant in <i>CEP78</i> , a founder allele associated with cone‐rod dystrophy, hearing loss, and reduced male fertility
De Novo <i>ARID1B</i> mutations cause growth delay associated with aberrant Wnt/β–catenin signaling
Fundamental role of BMP15 in human ovarian folliculogenesis revealed by null and missense mutations associated with primary ovarian insufficiency
Single‐molecule detection of cancer mutations using a novel PCR‐LDR‐qPCR assay
From genotype to phenotype: Early prediction of disease severity in argininosuccinic aciduria
Mutations in<i>FASTKD2</i>are associated with mitochondrial disease with multi‐OXPHOS deficiency
<i>POLD1</i> variants leading to reduced polymerase activity can cause hearing loss without syndromic features
Protein‐elongating mutations in<i>MYH11</i>are implicated in a dominantly inherited smooth muscle dysmotility syndrome with severe esophageal, gastric, and intestinal disease
Issue Information
A protein‐centric approach for exome variant aggregation enables sensitive association analysis with clinical outcomes
Genomics‐based treatment in a patient with two overlapping heritable skin disorders: Epidermolysis bullosa and acrodermatitis enteropathica
A novel gain‐of‐function mutation in <i>SCN5A</i> responsible for multifocal ectopic Purkinje‐related premature contractions
Front Cover, Volume 41, Issue 2
Reclassification of a frequent African‐origin variant from <i>PMS2</i> to the pseudogene <i>PMS2CL</i> African-origin
Osteoporosis genome‐wide association study variant c.3781 C&gt;A is regulated by a novel anti‐osteogenic factor miR‐345‐5p
Molecular insights into the mechanism of nonrecurrent <i>F8</i> structural variants: Full breakpoint characterization and bioinformatics of DNA elements implicated in the upmos
Loss of<i>RAD9B</i>impairs early neural development and contributes to the risk for human spina bifida
Further delineation of putative <i>ACTB</i> loss‐of‐function variants: A 4‐patient series
Mutation update: Variants of the CYB5R3 gene in recessive congenital methemoglobinemia
Genetic and phenotypic spectrum associated with IFIH1 gain‐of‐function
An intellectual disability‐associated missense variant in TRMT1 impairs tRNA modification and reconstitution of enzymatic activity
Elucidation of de novo small insertion/deletion biology with parent‐of‐origin phasing
Hermansky–Pudlak syndrome: Mutation update
Differences in patient ascertainment affect the use of gene‐specified ACMG/AMP phenotype‐related variant classification criteria: Evidence for <i>TP53</i>
A human minisatellite hosts an alternative transcription start site for <i>NPRL3</i> driving its expression in a repeat number‐dependent manner
Issue Information
Response to “Should <i>FANCL</i> heterozygous pathogenic variants be considered as potentially causative of primary ovarian insufficiency?”
Should <i>FANCL</i> heterozygous pathogenic variants be considered as potentially causative of primary ovarian insufficiency?
Cover, Volume 41, Issue 9
Cover, Volume 41, Issue 8