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Extracellular Vesicle–Mediated U1 snRNA Delivery Restores Aberrant Pre-mRNA Splicing in Human Cells

2026-01-06

Abstract excerpt

Splicing defects represent a significant class of human genetic disorders, yet strategies to directly correct aberrant splice-site recognition remain limited. The small nuclear RNA U1 plays a critical role in pre-messenger RNA splicing by base-pairing with the conserved 5′ splice-site ‘GU’ dinucleotide. Disruption of this interaction can lead to abnormal splicing or frameshift mutations, contributing to disease pa...

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Literature Corpus work
84c8afb7-881a-5a90-b27e-8979b287d082
DOI
10.20944/preprints202601.0231.v1
Open publication

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Extracellular Vesicle–Mediated U1 snRNA Delivery Restores Aberrant Pre-mRNA Splicing in Human CellsDOI 10.20944/preprints202601.0231.v1
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