AAV2-Mediated Transduction of the Mouse Retina After Optic Nerve Injury.

ABSTRACT:

Purpose

Gene therapy of retinal ganglion cells (RGCs) has promise as a powerful therapeutic for the rescue and regeneration of these cells after optic nerve damage. However, early after damage, RGCs undergo atrophic changes, including gene silencing. It is not known if these changes will deleteriously affect transduction and transgene expression, or if the therapeutic protein can influence reactivation of the endogenous genome.

Methods

Double-transgenic mice carrying a Rosa26-(LoxP)-tdTomato reporter, and a mutant allele for the proapoptotic Bax gene were reared. The Bax mutant blocks apoptosis, but RGCs still exhibit nuclear atrophy and gene silencing. At times ranging from 1 hour to 4 weeks after optic nerve crush (ONC), eyes received an intravitreal injection of AAV2 viru

SUBMITTER: Nickells RW 

PROVIDER: S-EPMC5716181 | biostudies-literature | 2017 Dec

REPOSITORIES: biostudies-literature