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Mor-Shaked, H.

Publications and source records attributed to Mor-Shaked, H..

2 recordsLinked to original sources

SeverYX: An ensemble framework for robust variant severity classification

Accurate interpretation of genetic variants in clinical genomics depends on reconciling multiple computational predictors that often disagree or provide incomplete evidence. We present SeverYX, a transparent, rule-based ensemble framework that consolidates conservation, splicing, and functional-impact predictors into a three-tier severity classification ('High', 'Medium', 'Low') through an effect-aware decision architecture. We benchmarked SeverYX against a curated ClinVar dataset of 270,208 MANE-transcript variants (86,161 pathogenic/likely pathogenic, 184,047 benign/likely benign), stratified by molecular consequence, review status, and haploinsufficiency. The baseline configuration achieved 97.6% concordance with ClinVar; at a fixed High cut, 90.0% of pathogenic and 2.0% of benign variants were assigned High. Concordance was highest for loss-of-function variants and lowest for non-coding and unclassified pathogenic variants, reflecting known gaps in current predictive tools. Systematic threshold optimization, combined with the addition of AlphaMissense and REVEL as missense predictors, raised overall concordance to 98.7%, reduced the benign High rate to 0.4% (at a cost of pathogenic High sensitivity falling to 88.3%), and improved the Matthews correlation coefficient from 0.895 to 0.908. These results show that SeverYX offers a transparent, reproducible and extensible approach for consolidating heterogeneous variant-effect evidence into clinically interpretable severity calls, and a scalable path for incorporating future predictive models into genomic interpretation workflows.

genetics↗

Mutations in MYLPF cause a novel segmental amyoplasia that manifests as distal arthrogryposis

We identified ten persons in six consanguineous families with Distal Arthrogryposis (DA) who had congenital contractures, scoliosis, and short stature. Exome sequencing revealed that each affected person was homozygous for one of two different rare variants (c.470G>T, p.(Cys157Phe) or c.469T>C, p.(Cys157Arg)) affecting the same residue of myosin light chain, phosphorylatable, fast skeletal muscle (MYLPF). In a seventh family, a c.487G>A, p.(Gly163Ser) variant in MYLPF arose de novo in a father, who transmitted it to his son. In an eighth family comprised of seven individuals with dominantly-inherited DA, a c.98C>T, p.(Ala33Val) variant segregated in all four persons tested. Variants in MYLPF underlie both dominant and recessively inherited DA. Mylpf protein models suggest that the residues associated with dominant DA interact with myosin whereas the residues altered in families with recessive DA only indirectly impair this interaction. Pathological and histological exam of a foot amputated from an affected child revealed complete absence of skeletal muscle (i.e., segmental amyoplasia). To investigate the mechanism for this finding, we generated an animal model for partial MYLPF impairment by knocking out zebrafish mylpfa. The mylpfa mutant had reduced trunk contractile force and complete pectoral fin paralysis, demonstrating that mylpf impairment most severely affects limb movement. mylpfa mutant muscle weakness was most pronounced in an appendicular muscle and was explained by reduced myosin activity and fiber degeneration. Collectively, our findings demonstrate that partial loss of MYLPF function can lead to congenital contractures, likely as a result of degeneration of skeletal muscle in the distal limb.

genetics↗