Title |
Comparative Analysis of DNA Nanoparticles and AAVs for Ocular Gene Delivery
|
---|---|
Published in |
PLOS ONE, December 2012
|
DOI | 10.1371/journal.pone.0052189 |
Pubmed ID | |
Authors |
Zongchao Han, Shannon M. Conley, Rasha Makkia, Junjing Guo, Mark J. Cooper, Muna I. Naash |
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. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 1 | 2% |
Unknown | 62 | 98% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Researcher | 13 | 21% |
Student > Ph. D. Student | 12 | 19% |
Student > Master | 11 | 17% |
Student > Doctoral Student | 3 | 5% |
Student > Bachelor | 3 | 5% |
Other | 10 | 16% |
Unknown | 11 | 17% |
Readers by discipline | Count | As % |
---|---|---|
Biochemistry, Genetics and Molecular Biology | 12 | 19% |
Agricultural and Biological Sciences | 12 | 19% |
Pharmacology, Toxicology and Pharmaceutical Science | 6 | 10% |
Medicine and Dentistry | 6 | 10% |
Neuroscience | 3 | 5% |
Other | 9 | 14% |
Unknown | 15 | 24% |