Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA <i>in vivo</i> induces homology-directed DNA repair.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 29805845.
- Also identified by DOI 10.1038/s41551-017-0137-2 and PMC identifier 5968829.
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Abstract
CRISPR/Cas9-based therapeutics, especially those that can correct gene mutations via homology directed repair (HDR), have the potential to revolutionize the treatment of genetic diseases. However, HDR-based therapeutics are challenging to develop because they require simultaneous <i>in vivo</i> delivery of Cas9 protein, guide RNA and donor DNA. Here, we demonstrate that a delivery vehicle composed of gold nanoparticles conjugated to DNA and complexed with cationic endosomal disruptive polymers can deliver Cas9 ribonucleoprotein and donor DNA into a wide variety of cell types, and efficiently correct the DNA mutation that causes Duchenne muscular dystrophy in mice via local injection, with minimal off-target DNA damage.