MicroRNA-122-mediated liver detargeting enhances the tissue specificity of cardiac genome editing

ABSTRACT: We use base editing to eliminate the CaMKII-delta, a major driver of heart disease. We found that insertion of microRNA122 target sequence (miR122 TS) in the 3' UTR of the vector eliminates editing in the liver, in combination with heart-specific promoters to specifically edit in heart.

ORGANISM(S): Mus musculus

PROVIDER: GSE245091 | GEO | 2025/10/10

REPOSITORIES: GEO