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PLoS One. 2012;7(12):e52189. doi: 10.1371/journal.pone.0052189. Epub 2012 Dec 18.

Comparative analysis of DNA nanoparticles and AAVs for ocular gene delivery.

Author information

1
Department of Cell Biology, University of Oklahoma Health Sciences Center, Oklahoma City, Oklahoma, United States of America.

Abstract

Gene therapy is a critical tool for the treatment of monogenic retinal diseases. However, the limited vector capacity of the current benchmark delivery strategy, adeno-associated virus (AAV), makes development of larger capacity alternatives, such as compacted DNA nanoparticles (NPs), critical. Here we conduct a side-by-side comparison of self-complementary AAV and CK30PEG NPs using matched ITR plasmids. We report that although AAVs are more efficient per vector genome (vg) than NPs, NPs can drive gene expression on a comparable scale and longevity to AAV. We show that subretinally injected NPs do not leave the eye while some of the AAV-injected animals exhibited vector DNA and GFP expression in the visual pathways of the brain from PI-60 onward. As a result, these NPs have the potential to become a successful alternative for ocular gene therapy, especially for the multitude of genes too large for AAV vectors.

PMID:
23272225
PMCID:
PMC3525534
DOI:
10.1371/journal.pone.0052189
[Indexed for MEDLINE]
Free PMC Article

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