4.6 Review

Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics

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

  1. Alfred P. Sloan Foundation
  2. University of Iowa Graduate College
  3. National GEM Consortium
  4. University of Iowa Department of Obstetrics and Gynecology Research Development Fund
  5. National Cancer Institute at the National Institutes of Health [5P30CA086862]
  6. Lyle and Sharon Bighley Chair of Pharmaceutical Sciences

向作者/读者索取更多资源

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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