Gene Portals: A Framework for Integrating Clinical, Functional, and Structural Evidence into Rare Disease Variant Classification
Tobias Brünger, Ilona KreyiD, Suyeon Kim, Chiara KlöckneriD, Scott J. MyersiD, Katrine M. JohanneseniD, Arthur StefanskiiD, Gary Taylor, Eduardo Pérez‐PalmaiD, Marie MacneeiD, Stéphanie SchorgeiD, Rebekka S. DahliD, Hongjie YuaniD, Riley E. PerszykiD, Sukhan Kim, Sunanjay BajajiD, Ingo HelbigiD, Jen Q. PaniD, Mark FarrantiD, Lonnie P. WollmuthiD, David J. A. WyllieiD, Erkin KurganoviD, David Báez-NietoiD, Sameer M. ZuberiiD, Christian M. BoßelmanniD, , Dennis LaliD
Abstract
Rare Mendelian disorders affect 300-400 million people globally. Although genetic testing has become widely adopted, gene-specific evidence for tailored variant interpretation remains scattered across resources. We present Gene Portals, a framework for gene-centered multimodal knowledge bases that co-localize expert-harmonized clinical data, functional assays, population variation, structural annotations and gene-specific ACMG/AMP specifications within a single resource. A modular interface integrates this unified evidence with VCEP-refined ACMG specifications to enable automated gene-specific variant classification, infer molecular mechanisms, and support cross-gene analyses. We demonstrate the frameworks utility across five Gene portals spanning eleven neurodevelopmental disorder-associated genes, integrating data from 4,423 individuals with 2,838 unique variants, 36,149 ClinVar submissions, and 1,044 expert-curated molecular readouts. By organizing evidence that is otherwise dispersed across multiple sources into a unified, queryable framework, the SCN, GRIN, CACNA1A, SATB2 and SLC6A1 Gene Portals became widely used community resources and provide an extensible template for standardized rare-disease variant interpretation and mechanism-aware discovery.
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