@article{10.1371/journal.pone.0052189, doi = {10.1371/journal.pone.0052189}, author = {Han, Zongchao AND Conley, Shannon M. AND Makkia, Rasha AND Guo, Junjing AND Cooper, Mark J. AND Naash, Muna I.}, journal = {PLOS ONE}, publisher = {Public Library of Science}, title = {Comparative Analysis of DNA Nanoparticles and AAVs for Ocular Gene Delivery}, year = {2012}, month = {12}, volume = {7}, url = {https://doi.org/10.1371/journal.pone.0052189}, pages = {1-11}, 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.}, number = {12}, }