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Editing specific DNA cytosine bases on the target strand using a modified Un1Cas12f1 system
Updated
Abstract
Essence
An engineered compact Cas12f cytosine base editor can selectively edit the target DNA strand.
Evidence
A platform-engineering study used modeling, mutagenesis, alanine scanning, and male mouse in vivo testing to build enUn1Cas12f1-derived target-strand miniCBE tools.
Caveat
The in vivo evidence was limited to male mice, and the abstract does not report therapeutic correction or long-term safety.
Simplified
Key numbers
63.18 ± 11.56%
Editing Efficiency in
Efficiency of the engineered in human cell line.
~2%
Rates for
rates associated with the variant.