Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration
Abstract 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 o...
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Main Authors: | Lewis E. Fry, Lauren Major, Ahmed Salman, Lucy A. McDermott, Jun Yang, Andrew J. King, Michelle E. McClements, Robert E. MacLaren |
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Format: | Article |
Language: | English |
Published: |
Nature Portfolio
2025-02-01
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Series: | Communications Biology |
Online Access: | https://doi.org/10.1038/s42003-025-07557-3 |
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