BioengineeringbioRxiv
Mitigating a TDP-43 proteinopathy by targeting ataxin-2 using RNA-targeting CRISPR effector proteins
M. A. Zeballos C., H. J. Moore, T. J. Smith…·T. Gaj
This study uses RNA-targeting CRISPR effector proteins to reduce TDP-43 pathology by targeting ataxin-2 in a mouse model, improving function and survival.
DOI 10.1038/s41467-023-42147-z