What is it about?
Scientists created a more powerful, tiny gene-editing tool called MiniCasUltra by improving a naturally small CRISPR protein (Un1Cas12f1). This new tool is much better than its predecessor: it edits genes much more effectively, causes few accidental cuts elsewhere in the DNA, and can target many more locations in the genome (using a broader "PAM" code). Its compact size is key – it fits inside a single virus (AAV), the standard delivery method for gene therapy. Researchers proved its potential by successfully editing two disease genes simultaneously in mouse livers and by treating a mouse model of a blinding eye disease (neovascular AMD) by targeting a crucial growth factor (VEGFA). This breakthrough combines powerful editing with practical delivery for gene therapy.
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Why is it important?
Engineered ultracompact CRISPR (MiniCasUltra) combines high efficiency, broad targeting, & low off-target effects. Its tiny size fits in a single AAV, enabling simultaneous dual-gene editing in liver & treating eye disease (AMD) in mice.
Perspectives
I hope MiniCasUltra will become an important addition to the mini CRISPR toolbox, a powerful one. We welcome inquiries about the work and the reagents.
Professor Yu Wang
Chinese Academy of Sciences
Read the Original
This page is a summary of: Engineered Un1Cas12f1 with boosted gene-editing activity and expanded genomic coverage, Proceedings of the National Academy of Sciences, August 2025, Proceedings of the National Academy of Sciences,
DOI: 10.1073/pnas.2501292122.
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