Article

OLIGONUCLEOTIDES TARGETING THE 3’ SPLICE SITE DOWNSTREAM OF A MICROEXON AS AN INNOVATIVE THERAPY FOR AUTISM

2024-10-25

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> Microexons are highly conserved and mostly neuronal-specific 3-27 nucleotide exons, enriched in genes linked to autism spectrum disorders (ASD). We have previously shown decreased inclusion of a neuronal specific 24 bp microexon (exon 4) of the translational regulator CPEB4 in brains of idiopathic ASD cases and that this leads to CPEB4 aggregation and subsequent under-expre...

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Literature Corpus work: 97bda889-5c95-58c5-b373-8cf0bb5e0c4f
DOI: 10.1101/2024.10.25.620162

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