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...
Main Authors: | , , , , , , , |
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Format: | Journal article |
Language: | English |
Published: |
Nature Research
2025
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