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Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations
Gold nanoparticles that passively deliver CRISPR machinery to blood repopulating cells have been developed and are shown to be capable of precise editing of multiple genetic loci of therapeutic interest without cytotoxicity or reduced fitness.
- Reza Shahbazi
- , Gabriella Sghia-Hughes
- & Jennifer E. Adair