PMC:2669177 / 25182-26268 JSONTXT

{"target":"http://pubannotation.org/docs/sourcedb/PMC/sourceid/2669177","sourcedb":"PMC","sourceid":"2669177","source_url":"https://www.ncbi.nlm.nih.gov/pmc/2669177","text":"This study demonstrates significant rescue of an ocular disease phenotype with a non-viral gene delivery method. Our data indicate that ocular delivery of compacted DNA-nanoparticles carrying Rds cDNA at P5 results in transgene expression that is: a) rapid-onset (starts at PI-2) and high with levels elevated up to four-fold above the endogenous, b) widely distributed in all photoreceptors, c) properly localized to the OSs of rods and cones, and d) persistently detected up to 4 months post- treatment without any obvious adverse side effects. Nanoparticle injection also improved expression of key photoreceptor-specific proteins known to be affected by the ongoing photoreceptor degeneration in the rds +/− retina. Notably, IRBP-NMP nanoparticles afforded significant and persistent restoration of both rod and cone function, with full-field cone ERG amplitudes approaching those seen in WT mice. Ultrastructural rescue in nanoparticle-injected eyes was similarly pronounced; at four months post- treatment, IRBP-NMP animals exhibited properly oriented OSs with well-aligned discs.","tracks":[]}