Title |
Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair
|
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Published in |
Nature Biomedical Engineering, October 2017
|
DOI | 10.1038/s41551-017-0137-2 |
Pubmed ID | |
Authors |
Kunwoo Lee, Michael Conboy, Hyo Min Park, Fuguo Jiang, Hyun Jin Kim, Mark A. Dewitt, Vanessa A. Mackley, Kevin Chang, Anirudh Rao, Colin Skinner, Tamanna Shobha, Melod Mehdipour, Hui Liu, Wen-chin Huang, Freeman Lan, Nicolas L. Bray, Song Li, Jacob E. Corn, Kazunori Kataoka, Jennifer A. Doudna, Irina Conboy, Niren Murthy |
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 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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Spain | 5 | 2% |
Japan | 4 | 2% |
Canada | 4 | 2% |
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Netherlands | 4 | 2% |
India | 3 | 1% |
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Demographic breakdown
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Scientists | 76 | 32% |
Science communicators (journalists, bloggers, editors) | 14 | 6% |
Practitioners (doctors, other healthcare professionals) | 6 | 3% |
Mendeley readers
Geographical breakdown
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Unknown | 949 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Student > Bachelor | 146 | 15% |
Researcher | 117 | 12% |
Student > Master | 85 | 9% |
Student > Doctoral Student | 44 | 5% |
Other | 114 | 12% |
Unknown | 238 | 25% |
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Engineering | 62 | 7% |
Medicine and Dentistry | 34 | 4% |
Other | 124 | 13% |
Unknown | 281 | 30% |