Evidence map›Paper›PMID 41569028›Full record

ArticleInvestigative ophthalmology & visual science2026

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

Yue Wan, Xiangxiang Liu, Xuejing Yan, Shen Wu, Yufei Teng, Luyi Han, Jingxue Zhang, Ningli Wang

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PubMed Full text at PMC Publisher
In one paragraph

Article in Investigative ophthalmology & visual science, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

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3 · Its place in the literature

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4 · The record

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5 · Who and what money

Authors and funding

8 authors.

Yue WanDepartment of Ophthalmology, Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing, China.
Xiangxiang LiuDepartment of Ophthalmology, Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing, China.
Xuejing YanBeijing Institute of Ophthalmology, Beijing Tongren Hospital, Beijing Key Laboratory of Intelligent Diagnosis Technology and Equipment for Optic Nerve-Related Eye Diseases, Capital Medical University, Beijing, China.
Shen WuBeijing Institute of Ophthalmology, Beijing Tongren Hospital, Beijing Key Laboratory of Intelligent Diagnosis Technology and Equipment for Optic Nerve-Related Eye Diseases, Capital Medical University, Beijing, China.
Yufei TengBeijing Institute of Ophthalmology, Beijing Tongren Hospital, Beijing Key Laboratory of Intelligent Diagnosis Technology and Equipment for Optic Nerve-Related Eye Diseases, Capital Medical University, Beijing, China.
Luyi HanBeijing Institute of Ophthalmology, Beijing Tongren Hospital, Beijing Key Laboratory of Intelligent Diagnosis Technology and Equipment for Optic Nerve-Related Eye Diseases, Capital Medical University, Beijing, China.
Jingxue ZhangBeijing Institute of Ophthalmology, Beijing Tongren Hospital, Beijing Key Laboratory of Intelligent Diagnosis Technology and Equipment for Optic Nerve-Related Eye Diseases, Capital Medical University, Beijing, China.
Ningli WangDepartment of Ophthalmology, Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

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 stress markers in TM cells in vivo, effectively preventing age-dependent IOP elevation, preserving aqueous humor outflow facility, and maintaining RGC survival and visual function in young Tg-MYOCP370L mice. In aged Tg-MYOCP370L mice, AAV-DJ-mediated MYOC silencing similarly lowered IOP and improved outflow facility. Conclusions: AAV-DJ-mediated MYOC silencing effectively alleviated glaucomatous pathology in Tg-MYOCP370L mice, highlighting its potential as a gene therapy strategy for myocilin-associated glaucoma.

Indexed as

Cytoskeletal ProteinsDependovirusEye ProteinsGene SilencingGenetic TherapyGlaucoma, Open-AngleGlycoproteinsAnimalsAqueous HumorDisease Models, AnimalGene Therapy AgentsGenetic VectorsIntraocular PressureMiceMice, TransgenicMyocilinCytoskeletal ProteinsEye ProteinsGlycoproteinsMyocilinRNA, Small Interfering

Identifiers

PMID41569028
PMCPMC12849824

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.