Tiny CRISPR enzyme boosts in-body gene editing for targeted therapies
TL;DR
NIH-backed researchers identified the small Al3Cas12f CRISPR enzyme and engineered the Al3Cas12f RKK variant to dramatically improve editing in human cells, raising efficiency from under 10% to over 80% (up to 90% in a key region). This enzyme's size allows packaging into AAV delivery vectors, a major step toward in-body gene therapies for diseases like cancer and ALS; next, researchers will test AAV-packaged delivery to move toward clinical use.
- NIH-funded breakthrough shrinks CRISPR for precision delivery in the body National Institutes of Health (.gov)
- Comparative characterization of Cas12f orthologs reveals mechanistic features underlying enhanced genome editing efficiency Nature
- Compact CRISPR system unlocks targeted in-body gene editing, with up to 90% efficiency Phys.org
- ‘Mini Molecular Scissors’ Could Enable Targeted Gene Editing Labcompare
- New bite-sized CRISPR molecule may open doors for therapeutic genome editing EurekAlert!
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