Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration

CRISPR-Cas13 systems have therapeutic promise for the precise correction of point mutations in RNA. Using adenosine deaminase acting on RNA (ADAR) effectors, A-I base conversions can be targeted using guide RNAs (gRNAs). We compare the Cas13 effectors PspCas13b and Cas13bt3 for the repair of the gen...

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Bibliographic Details
Main Authors: Fry, LE, Major, L, Salman, A, McDermott, LA, Yang, J, King, AJ, McClements, ME, MacLaren, RE
Format: Journal article
Language:English
Published: Nature Research 2025

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