Non-viral therapeutic approaches to ocular diseases: An overview and future directions.

ABSTRACT: Currently there are no viable treatment options for patients with debilitating inherited retinal degeneration. The vast variability in disease-inducing mutations and resulting phenotypes has hampered the development of therapeutic interventions. Gene therapy is a logical approach, and recent work has focused on ways to optimize vector design and packaging to promote optimized expression and phenotypic rescue after intraocular delivery. In this review, we discuss ongoing ocular clinical trials, which currently use viral gene delivery, but focus primarily on new advancements in optimizing the efficacy of non-viral gene delivery for ocular diseases. Non-viral delivery systems are highly customizable, allowing functionalization to improve cellular and nuclear uptake, bypassing cellular degrada

SUBMITTER: Zulliger R 

PROVIDER: S-EPMC4699668 | biostudies-literature | 2015 Dec

REPOSITORIES: biostudies-literature