Human Mutation - 2014

195 articles | Last updated: 2025-12-03 14:12:57
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Contents
What Identity Crisis? Rapidly Mutating Y-STRs Facilitate Differentiation of Males
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Deciphering the<i>cis</i>-Regulatory Landscape of<i>SOX9</i>Implicated in Craniofacial Development and Isolated Pierre Robin Sequence
Regulation of body and brain size: role of MAP4 and other centrosomal proteins
WGS and targetted sequencing in acute lymphoblastic leukemia identifies putative enhancer mutation associated with over-expression of a growth factor
Contents
Mutations in<i>SNRPB</i>, Encoding Components of the Core Splicing Machinery, Cause Cerebro-Costo-Mandibular Syndrome
MitImpact: an Exhaustive Collection of Pre-computed Pathogenicity Predictions of Human Mitochondrial Non-synonymous Variants
Cholesterol and Cognition: A Question of Balance?
The Fragile X Mouse is Cured, Now for the Patients
Contents
Gain-of-Function Mutation in STIM1 (P.R304W) Is Associated with Stormorken Syndrome
Variants in<i>CUL4B</i>are Associated with Cerebral Malformations
Preliminary Functional Assessment and Classification of<i>DEPDC5</i>Variants Associated with Focal Epilepsy
Functional Characterization and Classification of Frequent Low-Density Lipoprotein Receptor Variants
Heterozygous Deep-Intronic Variants and Deletions in<i>ABCA4</i>in Persons with Retinal Dystrophies and One Exonic<i>ABCA4</i>Variant
The Mutational Landscape in Pediatric Acute Lymphoblastic Leukemia Deciphered by Whole Genome Sequencing
An Augmented<i>ABCA4</i>Screen Targeting Noncoding Regions Reveals a Deep Intronic Founder Variant in Belgian Stargardt Patients
Simultaneous Analysis of Hundreds of Y-Chromosomal SNPs for High-Resolution Paternal Lineage Classification using Targeted Semiconductor Sequencing
Novel<i>SLC5A2</i>Variants Contribute to Renal Glucosuria in Chinese Families: Abnormal Expression and Dysfunction of Variant SLC5A2
Mutations in<i>COA6</i>cause Cytochrome<i>c</i>Oxidase Deficiency and Neonatal Hypertrophic Cardiomyopathy
Next Generation Sequencing Identifies a Novel Rearrangement in the<i>HBB</i>Cluster Permitting to-the-Base Characterization
Whole-Exome Sequencing Identifies a Variant in<i>TMEM132E</i>Causing Autosomal-Recessive Nonsyndromic Hearing Loss DFNB99
MAP4-Dependent Regulation of Microtubule Formation Affects Centrosome, Cilia, and Golgi Architecture as a Central Mechanism in Growth Regulation
Clinical Applications and Implications of Common and Founder Mutations in Indian Subpopulations
Identification of Splicing Defects Caused by Mutations in the Dysferlin Gene
Genetics and Pathogenesis of Autosomal Dominant Polycystic Kidney Disease: 20 Years On
De Novo Mutations in the Motor Domain of KIF1A Cause Cognitive Impairment, Spastic Paraparesis, Axonal Neuropathy, and Cerebellar Atrophy
Redefining Mutational Spectra via Updated Locus-specific Databases
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A Genetic Variant in the Seed Region of miR-4513 Shows Pleiotropic Effects on Lipid and Glucose Homeostasis, Blood Pressure, and Coronary Artery Disease
Identification and Characterization of Aberrant<i>GAA</i>Pre-mRNA Splicing in Pompe Disease Using a Generic Approach
Improved Locus-Specific Database for<i>OPA1</i>Mutations Allows Inclusion of Advanced Clinical Data
Disruption of the<i>SEMA3D</i>Gene in a Patient with Congenital Heart Defects
Capture of Somatic mtDNA Point Mutations with Severe Effects on Oxidative Phosphorylation in Synaptosome Cybrid Clones from Human Brain
Integrated Sequence Analysis Pipeline Provides One-Stop Solution for Identifying Disease-Causing Mutations
<i>GNAS</i> Mutations in Pseudohypoparathyroidism Type 1a and Related Disorders
Altered<i>GPM6A/M6</i>Dosage Impairs Cognition and Causes Phenotypes Responsive to Cholesterol in Human and<i>Drosophila</i>
Nonsense Mutation in Coiled-Coil Domain Containing 151 Gene (<i>CCDC151</i>) Causes Primary Ciliary Dyskinesia
Identification of Fragile X Syndrome Specific Molecular Markers in Human Fibroblasts: A Useful Model to Test the Efficacy of Therapeutic Drugs
Identification of Pathogenic Mechanisms of<i>COCH</i>Mutations, Abolished Cochlin Secretion, and Intracellular Aggregate Formation: Genotype-Phenotype Correlations in DFNA9 Deafness and Vestibular Dis
New Target Genes in Endometrial Tumors Show a Role for the Estrogen-Receptor Pathway in Microsatellite-Unstable Cancers
Mutation Update for UBE3A Variants in Angelman Syndrome
Identification of Large<i>NF1</i>Duplications Reciprocal to NAHR-Mediated Type-1<i>NF1</i>Deletions
Mosaicism for Dominant Collagen 6 Mutations as a Cause for Intrafamilial Phenotypic Variability
Mutation Update: The Spectra of Nebulin Variants and Associated Myopathies
Hidden Mutations in Cornelia de Lange Syndrome Limitations of Sanger Sequencing in Molecular Diagnostics
High Frequency of<i>RPL22</i>Mutations in Microsatellite-Unstable Colorectal and Endometrial Tumors
Mutations in<i>NTRK3</i>Suggest a Novel Signaling Pathway in Human Congenital Heart Disease
A New Overgrowth Syndrome is due to Mutations in<i>RNF125</i>
A CGG-Repeat Expansion Mutation in<i>ZNF713</i>Causes FRA7A: Association with Autistic Spectrum Disorder in two Families
RASopathy-Associated<i>CBL</i>Germline Mutations Cause Aberrant Ubiquitylation and Trafficking of EGFR
Three Different Cone Opsin Gene Array Mutational Mechanisms; Genotype-Phenotype Correlation and Functional Investigation of Cone Opsin Variants
Comprehensive Analysis of Pathogenic Deletion Variants in Fanconi Anemia Genes
Impaired Function is a Common Feature of Neuropathy-Associated Glycyl-tRNA Synthetase Mutations
Functional and Clinical Impact of Novel<i>Tmprss6</i>Variants in Iron-Refractory Iron-Deficiency Anemia Patients and Genotype-Phenotype Studies
An Efficient Pipeline for the Generation and Functional Analysis of Human<i>BRCA2</i>Variants of Uncertain Significance
Type I Procollagen C-Propeptide Defects: Study of Genotype-Phenotype Correlation and Predictive Role of Crystal Structure
A Novel<i>SHOC2</i>Variant in Rasopathy
Instability of Trinucleotidic Repeats During Chromatin Remodeling in Spermatids
Impaired Development Of Neural-Crest Cell Derived Organs and Intellectual Disability Caused By<i>MED13L</i>Haploinsufficiency
Morquio <scp>A</scp> Syndrome‐Associated Mutations: A Review of Alterations in the <i>GALNS</i> Gene and a New Locus‐Specific Database
A Mutation in the<i>CASQ1</i>Gene Causes a Vacuolar Myopathy with Accumulation of Sarcoplasmic Reticulum Protein Aggregates
Mutational and Functional Analysis of the Tumor-Suppressor PTPRD in Human Melanoma
Contents
Addressing Variant Pathogenicity: The TorsinA (<i>TOR1A</i>) Gene as a Model
Functional Characterization and Categorization of Missense Mutations that Cause Methylmalonyl‐ <scp>C</scp> o <scp>A</scp> Mutase ( <scp>MUT
Mutation in The Nuclear-Encoded Mitochondrial Isoleucyl-tRNA Synthetase<i>IARS2</i>in Patients with Cataracts, Growth Hormone Deficiency with Short Stature, Partial Sensorineural Deafness, and Periphe
Experimental Assessment of Splicing Variants Using Expression Minigenes and Comparison with In Silico Predictions
A New Workflow for Whole-Genome Sequencing of Single Human Cells
Genetic and Clinical Analysis of <i> <scp>ABCA</scp> 4 </i> ‐Associated Disease in African American Patients African American Patients
A Functional SNP Catalog of Overlapping miRNA-Binding Sites in Genes Implicated in Prion Disease and Other Neurodegenerative Disorders
Mutation of<i>POC1B</i>in a Severe Syndromic Retinal Ciliopathy
Diagnostic Exome Sequencing to Elucidate the Genetic Basis of Likely Recessive Disorders in Consanguineous Families
Online Registry for Mutations in Hereditary Amyloidosis Including Nomenclature Recommendations
Complex Tissue-Specific Epigenotypes in Russell-Silver Syndrome Associated with 11p15 ICR1 Hypomethylation
Evidence Against<i>RAB40AL</i>Being the Locus for Martin-Probst X-Linked Deafness-Intellectual Disability Syndrome
Functional Analyses of Mutations in<i>HEPACAM</i>Causing Megalencephalic Leukoencephalopathy
Gain‐of‐Function Mutation in STIM1 (P.R304W) Is Associated with Stormorken Syndrome
Insights into the Regulatory Domain of Cystathionine Beta-Synthase: Characterization of Six Variant Proteins
GALT Protein Database: Querying Structural and Functional Features of GALT Enzyme
Destruction of DDIT3/CHOP Protein by Wild-Type SPOP but Not Prostate Cancer-Associated Mutants
A Rising Titan:<i>TTN</i>Review and Mutation Update
Mutations in Exon 1 Highlight the Role of<i>MED12</i>in Uterine Leiomyomas
HSF4 Mutation p.Arg116His Found in Age-Related Cataracts and in Normal Populations Produces Childhood Lamellar Cataract in Transgenic Mice
RNA Sequencing of Creatine Transporter (SLC6A8) Deficient Fibroblasts Reveals Impairment of the Extracellular Matrix
Enrichment of LOVD-USHbases with 152<i>USH2A</i>Genotypes Defines an Extensive Mutational Spectrum and Highlights Missense Hotspots
Unraveling Cellular Phenotypes of Novel<i>TorsinA/TOR1A</i>Mutations
Biochemical and Cellular Analysis of Human Variants of the DYT1 Dystonia Protein, TorsinA/TOR1A
Deletions in the 3′ Part of the<i>NFIX</i>Gene Including a Recurrent Alu-Mediated Deletion of Exon 6 and 7 Account for Previously Unexplained Cases of Marshall-Smith Syndrome
Assessing How Reduced Expression Levels of the Mismatch Repair Genes<i>MLH1</i>,<i>MSH2</i>, and<i>MSH6</i>Affect Repair Efficiency
Palindrome-Related Mutations in Neurofibromatosis 1: a New Hot-Spot, at PATRR17
Corneal Dystrophy-Causing SLC4A11 Mutants: Suitability for Folding-Correction Therapy
Contents
Toward Male Individualization with Rapidly Mutating Y-Chromosomal Short Tandem Repeats
Majority Vote and Other Problems when using Computational Tools
Identification of Novel Craniofacial Regulatory Domains Located far Upstream of<i>SOX9</i>and Disrupted in Pierre Robin Sequence
Spectrum of the Mutations in Bernard-Soulier Syndrome
Ancient and Recent Adaptive Evolution in the Antiviral<i>TRIM22</i>Gene: Identification of a Single-Nucleotide Polymorphism That Impacts TRIM22 Function
A Novel in-Frame 18-bp Microdeletion in<i>MT-CYB</i>Causes a Multisystem Disorder with Prominent Exercise Intolerance
A Novel Homozygous Mutation in FGFR3 Causes Tall Stature, Severe Lateral Tibial Deviation, Scoliosis, Hearing Impairment, Camptodactyly, and Arachnodactyly
Genetic Variations and Diseases in UniProtKB/Swiss‐Prot: The Ins and Outs of Expert Manual Curation
Contents
Genetic and Epigenetic Determinants of Low Dysferlin Expression in Monocytes
The Challenge for the Next Generation of Medical Geneticists
Genetic and Epigenetic Characteristics of FSHD-Associated 4q and 10q D4Z4 that are Distinct from Non-4q/10q D4Z4 Homologs
Mutation Databases for Inherited Renal Disease: Are They Complete, Accurate, Clinically Relevant, and Freely Available?
<i>VARS2</i>and<i>TARS2</i>Mutations in Patients with Mitochondrial Encephalomyopathies
Reducing False-Positive Incidental Findings with Ensemble Genotyping and Logistic Regression Based Variant Filtering Methods
Structural Genomic Variation as Risk Factor for Idiopathic Recurrent Miscarriage
Mutation Update for<i>GNE</i>Gene Variants Associated with GNE Myopathy
Genetic Screening and Functional Characterization of<i>PDGFRB</i>Mutations Associated with Basal Ganglia Calcification of Unknown Etiology
Mutations in<i>ALDH1A3</i>Represent a Frequent Cause of Microphthalmia/Anophthalmia in Consanguineous Families
A Novel ZRS Mutation Leads to Preaxial Polydactyly Type 2 in a Heterozygous Form and Werner Mesomelic Syndrome in a Homozygous Form
Palindrome-Mediated and Replication-Dependent Pathogenic Structural Rearrangements within the<i>NF1</i>Gene
Functional Studies of Tyrosine Hydroxylase Missense Variants Reveal Distinct Patterns of Molecular Defects in <scp>Dopa</scp> ‐Responsive Dystonia
The<i>MTHFR</i>C677T/c.665C&gt;T Polymorphism and Malaria: A Selective Advantage?
Contents
Performance of Protein Disorder Prediction Programs on Amino Acid Substitutions
The<i>TP53</i>Gene Network in a Postgenomic Era
<i>TCIRG1</i>-Associated Congenital Neutropenia
Recommendations for Analyzing and Reporting<i>TP53</i>Gene Variants in the High-Throughput Sequencing Era
<i>TBC1D24</i>Mutation Causes Autosomal-Dominant Nonsyndromic Hearing Loss
A Dominant Mutation in the Stereocilia-Expressing Gene<i>TBC1D24</i>is a Probable Cause for Nonsyndromic Hearing Impairment
Germline<i>TP53</i>Mutations and the Changing Landscape of Li-Fraumeni Syndrome
Cantú Syndrome Resulting from Activating Mutation in the<i>KCNJ8</i>Gene
Analysis of TP53 Mutation Status in Human Cancer Cell Lines: A Reassessment
Mutation Update and Genotype-Phenotype Correlations of Novel and Previously Described Mutations in<i>TPM2</i>and<i>TPM3</i>Causing Congenital Myopathies
Cadherin 5 is Regulated by Corticosteroids and Associated with Central Serous Chorioretinopathy
TP53 Mutations in Human Cancer: Database Reassessment and Prospects for the Next Decade
Novel Mutations Widen the Phenotypic Spectrum of Slow Skeletal/β-Cardiac Myosin (<i>MYH7</i>) Distal Myopathy
<i>CDKN2A</i>Unclassified Variants in Familial Malignant Melanoma: Combining Functional and Computational Approaches for Their Assessment
<i>STK11</i>Domain XI Mutations: Candidate Genetic Drivers Leading to the Development of Dysplastic Polyps in Peutz-Jeghers Syndrome
New Clues to the Relationship Between Endoplasmic Reticulum, Lipid Metabolism and Axonopathies
Contents
The Challenge of Genomic Medicine
Molecular Analysis, Pathogenic Mechanisms, and Readthrough Therapy on a Large Cohort of<scp>K</scp>abuki Syndrome Patients
A Frameshift Mutation in<i>GRXCR2</i>Causes Recessively Inherited Hearing Loss
A Dominant STIM1 Mutation Causes Stormorken Syndrome
Identification of a Novel 5′ Alternative<i>CFTR</i>mRNA Isoform in a Patient with Nasal Polyposis and<i>CFTR</i>Mutations
An<i>FBN1</i>Deep Intronic Mutation in a Familial Case of Marfan Syndrome: An Explanation for Genetically Unsolved Cases?
A Robust Approach for Blind Detection of Balanced Chromosomal Rearrangements with Whole-Genome Low-Coverage Sequencing
Pathogenic: Light or Dark Skin?
Gene Conversion Violates the Stepwise Mutation Model for Microsatellites in<scp>Y</scp>‐Chromosomal Palindromic Repeats
Elucidating Common Structural Features of Human Pathogenic Variations Using Large-Scale Atomic-Resolution Protein Networks
Mutations in the Human<i>UBR1</i>Gene and the Associated Phenotypic Spectrum
Molecular Characterization and Cancer Risk Associated with BRCA1 and BRCA2 Splice Site Variants Identified in Multiple-Case Breast Cancer Families
The Genome Clinic: A Multidisciplinary Approach to Assessing the Opportunities and Challenges of Integrating Genomic Analysis into Clinical Care
The UMD-APC Database, a Model of Nation-Wide Knowledge Base: Update with Data from 3,581 Variations
An Evaluation of Copy Number Variation Detection Tools from Whole-Exome Sequencing Data
The Mechanism by which TATA-Box Polymorphisms Associated with Human Hereditary Diseases Influence Interactions with the TATA-Binding Protein
Targeted and Genomewide NGS Data Disqualify Mutations in<i>MYO1A</i>, the “<i>DFNA48</i>Gene”, as a Cause of Deafness
Increased Resistance to Malaria in Mice with Methylenetetrahydrofolate Reductase (<i>Mthfr</i>) Deficiency Suggests a Mechanism for Selection of the<i>MTHFR</i>677C&gt;T (c.665C&gt;T) Variant
A Recurrent Loss-of-Function Alanyl-tRNA Synthetase (<i>AARS</i>) Mutation in Patients with Charcot-Marie-Tooth Disease Type 2N (CMT2N)
Contents
Disease-causing Enhancer Variants: A Question of Penetrance
Jannovar: A Java Library for Exome Annotation
Genomics into Healthcare: The 5th Pan Arab Human Genetics Conference and 2013 Golden Helix Symposium
<i>TBC1D7</i>Mutations are Associated with Intellectual Disability, Macrocrania, Patellar Dislocation, and Celiac Disease
Integrating Massively Parallel Sequencing into Diagnostic Workflows and Managing the Annotation and Clinical Interpretation Challenge
Mutants TP53 p.R273H and p.R273C but not p.R273G Enhance Cancer Cell Malignancy
Expanding the Mutational Spectrum of<i>CRLF1</i>in Crisponi/CISS1 Syndrome
The Mdm Network and Its Regulation of p53 Activities: A Rheostat of Cancer Risk
How the<i>TP53</i>Family Proteins<i>TP63</i>and<i>TP73</i>Contribute to Tumorigenesis: Regulators and Effectors
Primary Immunodeficiency Caused by an Exonized Retroposed Gene Copy Inserted in the<i>CYBB</i>Gene
Functional Mutation Analysis Provides Evidence for a Role of REEP1 in Lipid Droplet Biology
Locus-Specific Databases in Cancer: What Future in a Post-Genomic Era? The TP53 LSDB paradigm
Prioritizing Disease-Linked Variants, Genes, and Pathways with an Interactive Whole-Genome Analysis Pipeline
Clinically Relevant Variants - Identifying, Collecting, Interpreting, and Disseminating: The 2013 Annual Scientific Meeting of the Human Genome Variation Society
Delineation of<i>EFTUD2</i>Haploinsufficiency-Related Phenotypes Through a Series of 36 Patients
Prediction of the Repeat Domain Structures and Impact of Parkinsonism-Associated Variations on Structure and Function of all Functional Domains of Leucine-Rich Repeat Kinase 2 (LRRK2)
TP53 Mutants in the Tower of Babel of Cancer Progression
Two Novel Mutations in the<i>BCKDK</i>(Branched-Chain Keto-Acid Dehydrogenase Kinase) Gene Are Responsible for a Neurobehavioral Deficit in Two Pediatric Unrelated Patients
Insights into Wild-Type and Mutant p53 Functions Provided by Genetically Engineered Mice
TP53 Mutation Analysis in Clinical Practice: Lessons From Chronic Lymphocytic Leukemia
Thirteen New Patients with Guanidinoacetate Methyltransferase Deficiency and Functional Characterization of Nineteen Novel Missense Variants in the<i>GAMT</i>Gene
Human Cell Line Model for Cancer-Associated<i>KRAS</i>Noncoding SNP
Contents
A Novel Splice Site Mutation in the Noncoding Region of<i>BRCA2</i>: Implications for Fanconi Anemia and Familial Breast Cancer Diagnostics
Mutant TP53 Posttranslational Modifications: Challenges and Opportunities
A New and More Accurate Estimate of the Rate of Concurrent Tandem-Base Substitution Mutations in the Human Germline: ∼0.4% of the Single-Nucleotide Substitution Mutation Rate
Mucolipidosis II-Related Mutations Inhibit the Exit from the Endoplasmic Reticulum and Proteolytic Cleavage of GlcNAc-1-Phosphotransferase Precursor Protein (<i>GNPTAB</i>)
<scp>DIAMUND</scp> : Direct Comparison of Genomes to Detect Mutations
Contribution of SUN1 Mutations to the Pathomechanism in Muscular Dystrophies
Novel<i>KCNQ2</i>and<i>KCNQ3</i>Mutations in a Large Cohort of Families with Benign Neonatal Epilepsy: First Evidence for an Altered Channel Regulation by Syntaxin-1A
Increased Dosage of RAB39B Affects Neuronal Development and Could Explain the Cognitive Impairment in Male Patients with Distal Xq28 Copy Number Gains
Early Frameshift Mutation in<i>PIGA</i>Identified in a Large XLID Family Without Neonatal Lethality
An Impairment of Long Distance SOX10 Regulatory Elements Underlies Isolated Hirschsprung Disease
Gender and Cell-Type-Specific Effects of the Transcription-Coupled Repair Protein, ERCC6/CSB, on Repeat Expansion in a Mouse Model of the Fragile X-Related Disorders
Tumor Heterogeneity Revealed by<i>KRAS</i>,<i>BRAF</i>, and<i>PIK3CA</i>Pyrosequencing:<i>KRAS</i>and<i>PIK3CA</i>Intratumor Mutation Profile Differences and Their Therapeutic Implications
Heterozygote PCR Product Melting Curve Prediction
Activating Mutations Cluster in the “Molecular Brake” Regions of Protein Kinases and Do Not Associate with Conserved or Catalytic Residues
Novel Dynein<i>DYNC1H1</i>Neck and Motor Domain Mutations Link Distal Spinal Muscular Atrophy and Abnormal Cortical Development
Robust Diagnostic Genetic Testing Using Solution Capture Enrichment and a Novel Variant‐Filtering Interface