Journal
NATURE BIOMEDICAL ENGINEERING
Volume 1, Issue 11, Pages 889-901Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/s41551-017-0137-2
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Funding
- National Institutes of Health [U01 268201000043C-0-0-1, R56 AI107116-01]
- Calico
- Roger's
- Strategies for Engineered Negligible Senescence Research Foundation
- W. M. Keck Foundation
- Moore Foundation
- Li Ka Shing Foundation
- Center of Innovation programme of the Japan Science and Technology Agency
- CIRM training grant [TG2-01164]
- National Institutes of Health S10 Instrumentation [S10RR029668, S10RR027303]
- Grants-in-Aid for Scientific Research [25000006] Funding Source: KAKEN
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Clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR associated protein 9 (Cas9)-based therapeutics, especially those that can correct gene mutations via homology-directed repair, have the potential to revolutionize the treatment of genetic diseases. However, it is challenging to develop homology-directed repair-based therapeutics because they require the simultaneous in vivo 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.
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