BioengineeringNature Biomedical EngineeringPeer-reviewed
In vivo CRISPR base editing for treatment of Huntington's disease
Shraddha Shirguppe, Michael Gapinske, Devyani Swami…·Pablo Pérez‐Piñera
This study uses CRISPR base editing to create HTT protein isoforms resistant to caspase-6 cleavage, reducing toxicity and neurodegeneration in a rodent HD model.
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