Human Mutation - 2023

34 articles | Last updated: 2025-12-03 14:12:57
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T A T A T A T A
Compendium of Clinical Variant Classification for 2,246 Unique ABCA4 Variants to Clarify Variant Pathogenicity in Stargardt Disease Using a Modified ACMG/AMP Framework
REVEL Is Better at Predicting Pathogenicity of Loss-of-Function than Gain-of-Function Variants
Evaluating the Utility of REVEL and CADD for Interpreting Variants in Amyotrophic Lateral Sclerosis Genes
Evaluating the Utility of a New Pathogenicity Predictor for Pediatric Cardiomyopathy
Clinical SMN1 and SMN2 Gene-Specific Sequencing to Enhance the Clinical Sensitivity of Spinal Muscular Atrophy Diagnostic Testing
Long-Read Sequencing Identified a Large Novel δ/β-Globin Gene Deletion in a Chinese Family Chinese; Quanzhou region of Southeast China; Chinese family
Genotype and Phenotype Characteristics of Chinese Pediatric Patients with Primary Hyperoxaluria Chinese; ethnicities
Combination of Synonymous and Missense Mutations in JAK3 Gene Contributes to Severe Combined Immunodeficiency in One Child
A Likelihood Ratio Approach for Utilizing Case-Control Data in the Clinical Classification of Rare Sequence Variants: Application to BRCA1 and BRCA2
The UCMD-Causing COL6A1 (<a:math xmlns:a="http://www.w3.org/1998/Math/MathML" id="M1"> <a:mi>c</a:mi> <a:mo>.</a:mo> <a:mn>930</a:mn>
Evaluation in Monogenic Diabetes of the Impact of GCK, HNF1A, and HNF4A Variants on Splicing through the Combined Use of In Silico Tools and Minigene Assays
Early-Onset Aortic Dissection: Characterization of a New Pathogenic Splicing Variation in the MYH11 Gene with Several In-Frame Abnormal Transcripts
Unexpected Inheritance Patterns in a Large Cohort of Patients with a Suspected Ciliopathy
Phenotypic Diversity in GNAO1 Patients: A Comprehensive Overview of Variants and Phenotypes
A Novel Constitutively Active <a:math xmlns:a="http://www.w3.org/1998/Math/MathML" id="M1"> <a:mi>c</a:mi> <a:mo>.</a:mo> <a:mn>98</a:mn> Japanese
The Common PKD1 p.(Ile3167Phe) Variant Is Hypomorphic and Associated with Very Early Onset, Biallelic Polycystic Kidney Disease
BTKbase, Bruton Tyrosine Kinase Variant Database in X-Linked Agammaglobulinemia: Looking Back and Ahead
Splicing Analysis of MYO5B Noncanonical Variants in Patients with Low Gamma-Glutamyltransferase Cholestasis
A Comprehensive LOVD Database for Fatty Acid Oxidation Disorders in Chinese Populations
Targeted Genomic Sequencing of TSC1 and TSC2 Reveals Causal Variants in Individuals for Whom Previous Genetic Testing for Tuberous Sclerosis Complex Was Normal
Functional Characterization of Novel Lunatic Fringe Variants in Spondylocostal Dysostosis Type-III with Scoliosis
Disruption of Intracellular Calcium Homeostasis Leads to ERLIN2-Linked Hereditary Spastic Paraplegia in Patient-Derived Stem Cell Models
The Spectra of Disease-Causing Mutations in the Ferroportin 1 (SLC40A1) Encoding Gene and Related Iron Overload Phenotypes (Hemochromatosis Type 4 and Ferroportin Disease)
Balanced Translocation Disrupting JAG1 Identified by Optical Genomic Mapping in Suspected Alagille Syndrome Han Chinese
Frequency and Functional Characterization of RUNX1 Germline Variants in Myeloid Neoplasms
Reasons and Resolutions for Inconsistent Variant Interpretation
The Broad Spectrum of TP53 Mutations in CLL: Evidence of Multiclonality and Novel Mutation Hotspots
Quantitative Phenotype Morbidity Description of SATB2-Associated Syndrome
Somatic Double Inactivation of NF1 Associated with NF1-Related Pectus Excavatum Deformity
A Palindrome-Like Structure on 16p13.3 Is Associated with the Formation of Complex Structural Variations and SRRM2 Haploinsufficiency
A Novel Alu Element Insertion in ATM Induces Exon Skipping in Suspected HBOC Patients
Large Region of Homozygous (ROH) Identified in Indian Patients with Autosomal Recessive Limb-Girdle Muscular Dystrophy with p.Thr182Pro Variant in SGCB Gene West Eurasian/European ancestry
Specifications of the ACMG/AMP Variant Classification Guidelines for Germline DICER1 Variant Curation
Functional Assays Combined with Pre-mRNA-Splicing Analysis Improve Variant Classification and Diagnostics for Individuals with Neurofibromatosis Type 1 and Legius Syndrome