Journal
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
Volume 54, Issue 41, Pages 12029-12033Publisher
WILEY-V C H VERLAG GMBH
DOI: 10.1002/anie.201506030
Keywords
CRISPR-Cas9; DNA; drug delivery; genome editing; nanoparticles
Categories
Funding
- NC TraCS
- NIH (Clinical and Translational Science Awards CTSA) [1L1TR001111]
- NSF [MCB-1452902]
- Direct For Biological Sciences
- Div Of Molecular and Cellular Bioscience [1452902] Funding Source: National Science Foundation
Ask authors/readers for more resources
CRISPR-Cas9 represents a promising platform for genome editing, yet means for its safe and efficient delivery remain to be fully realized. A novel vehicle that simultaneously delivers the Cas9 protein and single guide RNA (sgRNA) is based on DNA nanoclews, yarn-like DNA nanoparticles that are synthesized by rolling circle amplification. The biologically inspired vehicles were efficiently loaded with Cas9/sgRNA complexes and delivered the complexes to the nuclei of human cells, thus enabling targeted gene disruption while maintaining cell viability. Editing was most efficient when the DNA nanoclew sequence and the sgRNA guide sequence were partially complementary, offering a design rule for enhancing delivery. Overall, this strategy provides a versatile method that could be adapted for delivering other DNA-binding proteins or functional nucleic acids.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available