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Article

Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of <i>HNRNPH2</i> -related neurodevelopmental disorder

2025-11-05

Abstract excerpt

<h4>Summary</h4> Mutations in HNRNPH2 cause an X-linked disorder characterized by developmental delay, intellectual disability, motor and gait disturbances, and seizures. Murine models that reproduce key clinical features of HNRNPH2-related neurodevelopmental disorder suggest that it may result from a toxic gain of function of the mutant protein or a complex loss of normal HNRNPH2 function with impaired compensa...

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Literature Corpus work
b7a15aa1-fc88-5fd3-bc01-d64e694b4334
DOI
10.1101/2025.11.04.686541
Open publication

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Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of <i>HNRNPH2</i> -related neurodevelopmental disorderDOI 10.1101/2025.11.04.686541
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