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Article

Characterisation of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina

2024-02-11

Abstract excerpt

CRISPR-Cas13 nucleases are programmable RNA-targeting effectors that can silence gene expression in a reversible manner. Recent iterations of Cas13 nucleases are compact for adeno-associated virus (AAV) delivery to achieve strong and persistent expression in various organs in a safe manner. Here, we report significant transcriptomic signatures of Cas13bt3 expression in retinal cells and show all-in-one AAV gene th...

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Identifiers and source

Literature Corpus work
0abeab04-e39d-5de7-a7eb-99485f67cb93
DOI
10.1101/2024.02.10.579778
Open publication

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Characterisation of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retinaDOI 10.1101/2024.02.10.579778
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