期刊
NATURE
卷 540, 期 7631, 页码 144-+出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nature20565
关键词
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资金
- UCSD [P30 NS047101]
- CONACYT of Mexico
- 973 Program [2013CB967504, 2015CB964600]
- 863 Program [2014AA021604]
- Nomis Foundation
- Salk Women & Science Special Award
- NSFC [81601872]
- National Basic Research Program of China (973 Program) [2013CB967504, 2015CB964800, 2014CB910503]
- National Natural Science Foundation of China [81625009, 81371342, 81271266]
- National High Technology Research and Development Program of China [2014AA021604, 2015AA020307]
- Beijing Municipal Science and Technology Commission [Z151100003915072]
- RIKEN
- NIH [R01HL123755]
- King Abdullah University of Science and Technology (KAUST) Office of Sponsored Research (OSR) [OSR-2015-CRG4-2631]
- Leona M. and Harry B. Helmsley Charitable Trust [2012-PG-MED002]
- G. Harold and Leila Y. Mathers Charitable Foundation
- McKnight Foundation
- Moxie Foundation
- Fundacion Dr. Pedro Guillen
- Universidad Catolica San Antonio de Murcia (UCAM)
Targeted genome editing via engineered nucleases is an exciting area of biomedical research and holds potential for clinical applications. Despite rapid advances in the field, in vivo targeted transgene integration is still infeasible because current tools are inefficient(1), especially for non-dividing cells, which compose most adult tissues. This poses a barrier for uncovering fundamental biological principles and developing treatments for a broad range of genetic disorders(2). Based on clustered regularly interspaced short palindromic repeat/Cas9 (CRISPR/Cas9)(3,4) technology, here we devise a homology-independent targeted integration (HITI) strategy, which allows for robust DNA knock-in in both dividing and non-dividing cells in vitro and, more importantly, in vivo (for example, in neurons of postnatal mammals). As a proof of concept of its therapeutic potential, we demonstrate the efficacy of HITI in improving visual function using a rat model of the retinal degeneration condition retinitis pigmentosa. The HITI method presented here establishes new avenues for basic research and targeted gene therapies.
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