期刊
AAPS JOURNAL
卷 20, 期 6, 页码 -出版社
SPRINGER
DOI: 10.1208/s12248-018-0267-9
关键词
CRISPR; Cas9; gene editing; gene delivery; nanoparticle
资金
- Alfred P. Sloan Foundation
- University of Iowa Graduate College
- National GEM Consortium
- University of Iowa Department of Obstetrics and Gynecology Research Development Fund
- National Cancer Institute at the National Institutes of Health [5P30CA086862]
- Lyle and Sharon Bighley Chair of Pharmaceutical Sciences
The recent progress in harnessing the efficient and precise method of DNA editing provided by CRISPR/Cas9 is one of the most promising major advances in the field of gene therapy. However, the development of safe and optimally efficient delivery systems for CRISPR/Cas9 elements capable of achieving specific targeting of gene therapy to the location of interest without off-target effects is a primary challenge for clinical therapeutics. Nanoparticles (NPs) provide a promising means to meet such challenges. In this review, we present the most recent advances in developing innovative NP-based delivery systems that efficiently deliver CRISPR/Cas9 constructs and maximize their effectiveness.
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