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GR.1 Different functional consequences result in different phenotypes in CLCN4-related developmental and epileptic encephalopathy

Published online by Cambridge University Press:  05 June 2023

AN Sahly
Affiliation:
(Montreal)*
RE Guzman
Affiliation:
(Jülich)
J Sierra-Marquez
Affiliation:
(Jülich)
S Bungert-Plümke
Affiliation:
(Jülich)
A Franzen
Affiliation:
(Jülich)
L Mougharbel
Affiliation:
(Montreal)
S Berrahmoune
Affiliation:
(Montreal)
C Dassi
Affiliation:
(Montreal)
M Srour
Affiliation:
(Montreal)
KA Myers
Affiliation:
(Montreal)
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Abstract

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Background: Variants in CLCN4 are implicated in neurodevelopmental disorder, X-linked intellectual disability, and epileptic encephalopathy. CLCN4 encodes ClC-4, which is hypothesized to play a role in ion homeostasis and intracellular trafficking. ClC-4 relies on its formation of heterodimers with ClC-3, which possesses signals for target organelles. Methods: Case-Series. Then, we performed heterologous expression, patch-clamp electrophysiology, confocal microscopy, and protein biochemistry experiments to characterize our patients’ ClC-4 variants. Results: All three male patients had developmental and epileptic encephalopathy. Patients #1 and #2 had normal-appearing brains on MRI and no dysmorphic features. Patient #3 had: microcephaly, microsomia, complete agenesis of the corpus-callosum; and, cerebellar and brainstem hypoplasia. Patient #1 had recurrent status epilepticus separated by months of seizure freedom, while Patient #2 and #3 had brief, daily seizures. The p.Gly342Arg variant impaired the heterodimerization capability of ClC-4. The p.Ile549Leu and p.Asp89Asn variants exhibited early transport-activation, with p.Asp89Asn favouring higher transport-activity of ClC-4. Conclusions: We extend the phenotypic spectrum of CLCN4 variants and demonstrate the pathological functional-consequences of three previously unclassified variants. The p.Gly342Arg variant lead to a loss-of-function phenotype; however, the p.Ile549Leu and p.Asp89Asn variants likely caused gain-of-function phenotypes. Targeted animal or induced pluripotent stem-cell models are needed to further understand epileptogenic mechanisms of CLCN4 variants.

Type
Abstracts
Copyright
© The Author(s), 2023. Published by Cambridge University Press on behalf of Canadian Neurological Sciences Federation