Article

Allele-specific antisense oligonucleotide treatment rescues <i>atad3-</i> associated phenotype in zebrafish

2026-05-23

Abstract excerpt

<h4>ABSTRACT</h4> Pathogenic variants in ATAD3A cause a spectrum of multisystem disorders, with a recurrent dominant-negative variant (c.1582C>T; p.Arg528Trp) associated with neurodevelopmental disease. Given the tolerance of ATAD3A to heterozygous loss of function variants, allele-specific transcript reduction represents a promising therapeutic strategy. We designed and optimized allele-specific antisense olig...

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Literature Corpus work: e88e3dce-ed3c-5ab9-a167-1712f270e89e
DOI: 10.64898/2026.05.20.726050

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