AAV-DJ-Mediated MYOC Silencing as a Gene Therapy Approach for Myocilin-Associated Glaucoma.

ABSTRACT:

Purpose

To evaluate the therapeutic efficacy of an adeno-associated virus serotype DJ (AAV-DJ) vector delivering MYOC-targeting short-hairpin RNA (shMYOC) in a MYOCP370L transgenic glaucoma mouse model (Tg-MYOCP370L) for the treatment of open-angle glaucoma (OAG) associated with MYOC mutations.

Methods

An AAV-DJ vector, selected for its high transduction efficiency and tropism for trabecular meshwork (TM), was used to deliver shMYOC via a transpupillary intravitreal approach in Tg-MYOCP370L mice. Post-treatment evaluations included myocilin accumulation, ER stress marker expression, intraocular pressure (IOP), aqueous humor outflow facility, retinal ganglion cell (RGC) survival, and visual function.

Results

AAV-DJ-shMYOC markedly reduced myocilin accumulation and ER s

SUBMITTER: Wan Y 

PROVIDER: S-EPMC12849824 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature