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Aaron A Smargon

7
Publications
4
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Publications

Dual-targeting snRNA gene therapy rescues STMN2 and UNC13A splicing in TDP-43 proteinopathies.
bioRxiv : the preprint server for biology · 2025
Enhancing RNA base editing on mammalian transcripts with small nuclear RNAs.
Nature chemical biology · 2025
Small nuclear RNAs enhance protein-free RNA-programmable base conversion on mammalian coding transcripts.
bioRxiv : the preprint server for biology · 2024
Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders.
RNA (New York, N.Y.) · 2023
Engineered U1 snRNAs to modulate alternatively spliced exons.
Methods (San Diego, Calif.) · 2022
Crosstalk between CRISPR-Cas9 and the human transcriptome.
Nature communications · 2022
RNA-targeting CRISPR systems from metagenomic discovery to transcriptomic engineering.
Nature cell biology · 2020