Direct gene transfer with compacted DNA nanoparticles in retinal pigment epithelial cells: expression, repeat delivery and lack of toxicity.

ABSTRACT:

Aim

To evaluate the safety of compacted DNA nanoparticles (NPs) in retinal pigment epithelial (RPE) cells.

Materials & methods

Enhanced GFP expression cassettes controlled by the RPE-specific vitelloform macular dystrophy promoter were constructed with and without a bacterial backbone and compacted into NPs formulated with polyethylene glycol-substituted lysine 30-mers. Single or double subretinal injections were administered in adult BALB/c mice. Expression levels of enhanced GFP, proinflammatory cytokines and neutrophil/macrophage mediators, and retinal function by electroretinogram were evaluated at different time-points postinjection.

Results

Immunohistochemistry and real-time PCR demonstrated that NPs specifically transfect RPE cells at a higher efficiency than n

SUBMITTER: Han Z 

PROVIDER: S-EPMC3404893 | biostudies-literature | 2012 Apr

REPOSITORIES: biostudies-literature