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Engineered IscB-ωRNA system with a wider range of targets for base editing
Updated
Abstract
Ten of 19 uncharacterized IscB proteins from uncultured microbes showed activity in mammalian cells.
- The narrow targeting scope of IscB systems necessitates the identification of additional IscBs that recognize different target-adjacent motifs ().
- IscB ortholog IscB.m16 was enhanced through protein and ωRNA engineering, expanding its TAM scope from MRNRAA to NNNGNA.
- A variant named IscB.m16* was created, demonstrating improved targeting capabilities.
- IscB.m16*-derived base editors showed robust base-editing efficiency in mammalian cells.
- These base editors effectively restored Duchenne muscular dystrophy proteins in diseased mice using single adeno-associated virus delivery.
Simplified
Key numbers
46.15% ± 4.08%
Base-editing Efficiency Increase
Base-editing efficiency in HEK293T cells for *-.
NNNGNA
Expanded Range
range achieved by * compared to the previous MRNRAA.
3
Restoration of Expression
expression restoration in DMD mice after treatment.