GeneticsMolecular TherapyPeer-reviewed
Co-opting regulation bypass repair (CRBR) as a gene correction strategy for monogenic diseases
Jingjie Hu, Rebecca A. Bourne, Barbara C. McGrath…·Douglas R. Cavener
The paper introduces CRBR, a gene correction strategy that repairs mutations in both mitotic and post-mitotic cells, demonstrated in a mouse model of Wolcott-Rallison syndrome.
2 citations