Evidence map›Paper›PMID 40815429›Full record

ArticleNeurogenetics2025

Pathogenic KCNH2 variant in monozygotic twins with speech delay and lower risk type 2 long QT syndrome.

Katia Margiotti, Marco Fabiani, Costanza Zangheri, Antonella Cima, Francesca Monaco, Francesco Libotte, Chiara Ali', Maria Antonietta Barone, Antonella Viola, Alvaro Mesoraca and 1 more

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Article in Neurogenetics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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5 · Who and what money

Authors and funding

11 authors.

Katia Margiotti *Human Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy. katia.margiotti@artemisia.it.
Marco Fabiani *Human Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy.
Costanza ZangheriHuman Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy.
Antonella CimaHuman Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy.
Francesca MonacoHuman Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy.
Francesco LibotteHuman Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy.
Chiara Ali'Human Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy.
Maria Antonietta BaroneHuman Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy.
Antonella ViolaHuman Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy.
Alvaro MesoracaHuman Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy.
Claudio GiorlandinoHuman Genetics Lab, Altamedica Main Centre, Viale Liegi 45, Rome, 00198, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Type 2 Long QT Syndrome (type 2 LQTS) is a cardiac channelopathy caused by pathogenic variants in the KCNH2 gene, often associated with delayed cardiac repolarization and increased risk of arrhythmias. While its impact is traditionally considered cardiac, emerging studies suggest a potential role of KCNH2 dysfunction in neurogical disorders. We describe monozygotic twin sisters carrying the pathogenic frameshift variant KCNH2 c.2959_2960delCT (p.Leu987Valfs*131; rs748706373), inherited from their asymptomatic father. Clinically, both twins presented with severe language delay, absence of pointing, impaired social interaction, and stereotyped behaviors features consistent with neurogical disorders. Diagnostic diagnostic testing included whole exome sequencing (WES), chromosomal microarray (aCGH), and Fragile X screening. The KCNH2 variant emerged as the sole clinically significant finding. Cardiac evaluation through ECG and 24-hour Holter monitoring revealed no significant QT prolongation or arrhythmic episodes in either the twins or their father. No history of syncope, seizures, or cardiac events was reported. This report supports the variable expressivity and incomplete penetrance of KCNH2 variants in type 2 LQTS and raises the possibility that KCNH2 dysfunction may contribute to neurogical phenotypes manifestations. Causality remains to be established between KCNH2 and neurologic disorders. Though whole-genome sequencing remains to be completed in this pedigree, the potential association between KCNH2 and neurologic disorders is strengthened by the unique monozygotic presentation and the absence of known perinatal complications. Further studies are needed to clarify the association between KCNH2 variants and their contribution to neurological disorders, either through direct neural effects or indirectly via unrecognized perinatal arrhythmic events.

Indexed as

ERG1 Potassium ChannelLanguage Development DisordersLong QT SyndromeExome SequencingFemaleHumansMalePedigreeTwins, MonozygoticERG1 Potassium ChannelKCNH2 protein, humanKCNH2 geneMonozygotic twinsNeurodevelopmental disorders (NDDs)Speech delayType 2 long QT syndrome

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.