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Updated: Dec 5, 2025

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
Published on: February 17, 2023
Base editing: advances and therapeutic opportunities
Elizabeth M Porto1, Alexis C Komor2, Ian M Slaymaker3
1Department of Chemistry and Biochemistry, University of California, San Diego, La Jolla, CA, USA.
Abstract:
Base editing - the introduction of single-nucleotide variants (SNVs) into DNA or RNA in living cells - is one of the most recent advances in the field of genome editing. As around half of known pathogenic genetic variants are due to SNVs, base editing holds great potential for the treatment of numerous genetic diseases, through either temporary RNA or permanent DNA base alterations. Recent advances in the specificity, efficiency, precision and delivery of DNA and RNA base editors are revealing exciting therapeutic opportunities for these technologies. We expect the correction of single point mutations will be a major focus of future precision medicine.
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