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An Author Correction to this article was published on 01 May The delivery of CRISPR ribonucleoproteins RNPs for genome editing in vitro and in vivo has important advantages over other delivery methods, including reduced off-target and immunogenic effects.
However, effective delivery of RNPs remains challenging in certain cell types due to low efficiency and cell toxicity. To address these issues, we engineer self-deliverable RNPs that can promote efficient cellular uptake and carry out robust genome editing without the need for helper materials or biomolecules.
Further engineering of these fusion proteins establishes a C-terminal Cas9 fusion with three copies of A22p, a peptide derived from human semaphorin-3a, that exhibits substantially improved editing efficacy compared to other constructs. We find that self-deliverable Cas9 RNPs generate robust genome edits in clinically relevant genes when injected directly into the mouse striatum.
Overall, self-deliverable Cas9 proteins provide a facile and effective platform for genome editing in vitro and in vivo. CRISPR-Cas technology, adapted from bacterial immune systems, has been a revolutionary molecular tool for genome engineering in cells and organisms 1 , 2.
