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Article

High-throughput screens identify genotype-specific therapeutics for channelopathies.

JCI insight - 24 Nov 2025

Egly Christian L, Shen Alex, Do Tri Q, Tellet Cabiya Carlos, Ritschel Paxton A, Woo Suah, Ku Matthew, Delisle Brian P, Kroncke Brett M, Knollmann Björn C

Abstract excerpt

Genetic diseases such as ion channelopathies substantially burden human health. Existing treatments are limited and not genotype specific. Here, we report a 2-step high-throughput approach to rapidly identify drug candidates for repurposing as genotype-specific therapy. We first screened 1,680 medicines using a thallium-flux trafficking assay against Kv11.1 gene variants causing long QT syndrome (LQTS), an ion...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 41026531
DOI: 10.1172/jci.insight.191697
PMCID: PMC12643509

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

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