Research Summary: Comparative Analysis of DNA Nanoparticles and AAVs for Ocular Gene Delivery

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.

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Publisher: Public Library of Science

Date Published: 18-December-2012

Author(s): Han Z., Conley S., Makkia R., Guo J., Cooper M., Naash M.

Link: https://doi.org/10.1371/journal.pone.0052189

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