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Antisense oligonucleotides as a precision therapy for developmental and epileptic encephalopathies

1 Nov 2024

Abstract excerpt

Developmental and epileptic encephalopathies (DEEs) comprise a complex spectrum of neurological disorders characterized by neurodevelopmental delay and early-onset seizures primarily caused by diverse genetic mutations. Traditional treatments have largely been symptomatic, focusing on seizure control without addressing the underlying genetic causes. The advent of gene therapy, particularly through antisense...

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PMID: 39526481
DOI: 10.1111/cns.70050

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