292-PERT for Disease-Agnostic Nonsense Mutation Readthrough
The paper introduces a disease-agnostic genome-editing strategy called Prime Editing-mediated Readthrough of Premature Termination Codons (PERT). This technique uses prime editing to permanently convert a redundant endogenous transfer RNA (tRNA) into an optimized suppressor tRNA (sup-tRNA) to rescue diseases caused by premature stop codons (nonsense mutations). The authors detail their iterative screening process to identify the most potent sup-tRNA variants and optimize the prime editing components for efficient, one-time genomic installation. Crucially, they demonstrate that this approach…
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