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Peer-reviewed veterinary case report

Establishing a Zebrafish Functional Assay to Assess the Pathogenicity of Variants of Uncertain Significance in Ciliopathies.

Journal:
European journal of clinical investigation
Year:
2026
Authors:
Aresi, Carla et al.
Affiliation:
Department of Molecular Medicine · Italy

Abstract

A zebrafish morpholino knockdown model targeting ahi1 enables efficient phenotypic assessment of ciliopathy-related defects and functional evaluation of variants of uncertain significance. This assay clarifies the impact of VUSs, supporting zebrafish morphants as a reliable platform for validating ciliopathy-associated genetic variants.

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Original publication: https://pubmed.ncbi.nlm.nih.gov/42116549/