ArticleMolecular therapy : the journal of the American Society of Gene Therapy2017
Improved Intravitreal AAV-Mediated Inner Retinal Gene Transduction after Surgical Internal Limiting Membrane Peeling in Cynomolgus Monkeys.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 55 papers, 3 of them syntheses that pooled it.
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Who cites it
55 citing papers in PubMed, 3 syntheses or guidelines pooled it, 95 citations in OpenAlex.
- AAV Gene Therapy Drug Development and Translation of Engineered Ocular and Neurotropic Capsids: A Systematic Review Using Natural Language Processing.Clinical and translational science · 2025Pooled it
- Retinal Viral Gene Therapy: Impact of Route of Administration on Serious Adverse Events-A Systematic Review.Clinical & experimental ophthalmology · 2025Pooled it
- Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays.International journal of molecular sciences · 2020Pooled it
- Phase I/IIa Study of an Intravitreal Optogenetic Therapy (AGN-151597) in Patients with Advanced Retinitis Pigmentosa.Ophthalmology science · 2026Article
- The internal limiting basement membrane inhibits functional engraftment of transplanted human retinal ganglion cells in vivo.Science translational medicine · 2026Article
- AAV2-Retro-Mediated Gene Transfer Selectively Targets Outer Retinal Cells Following Intravitreal Injection.Investigative ophthalmology & visual science · 2026Article
- Photodisruption of the Inner Limiting Membrane Promotes Retinal Engraftment of Stem-Cell Derived Retinal Ganglion Cells.Investigative ophthalmology & visual science · 2026Article
- Laser photocoagulation improves the transduction efficiency of lentivirus vectors injected intravitreally.International journal of ophthalmology · 2026Article
- Inner Limiting Membrane Peel Extends In Vivo Calcium Imaging of Retinal Ganglion Cell Activity Beyond the Fovea in Non-Human Primate.Investigative ophthalmology & visual science · 2025Article
- Recent advancements in polymer science for retinal diseases: New frontiers in drug delivery systems.APL bioengineering · 2025Article
- Long-Term Diabetic Retinopathy Treatment Using Silicon Nanoneedles.Small (Weinheim an der Bergstrasse, Germany) · 2025Article
- Intravitreal AAV2 gene delivery to feline retinal ganglion cells.Vision research · 2025Article
- Gene therapy for glaucoma: Targeting key mechanisms.Vision research · 2024Review
- A Comparative Analysis of Models for AAV-Mediated Gene Therapy for Inherited Retinal Diseases.Cells · 2024Review
- AAV2-antiVEGFscFv gene therapy for retinal neovascularization.Molecular therapy. Methods & clinical development · 2023Article
- In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans.Molecular therapy. Methods & clinical development · 2023Article
- X-Linked Retinoschisis.Cold Spring Harbor perspectives in medicine · 2023Review
- Adeno-associated virus mediated gene therapy for neuroprotection of retinal ganglion cells in glaucoma.Vision research · 2023Review
- Bruch's Membrane: A Key Consideration with Complement-Based Therapies for Age-Related Macular Degeneration.Journal of clinical medicine · 2023Review
- Gene Therapy Activates Retinal Pigment Epithelium Cell Proliferation for Age-related Macular Degeneration in a Mouse Model.Current medical science · 2023Article
Corrections and comments
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Authors and funding
13 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The retina is an ideal target for gene therapy because of its easy accessibility and limited immunological response. We previously reported that intravitreally injected adeno-associated virus (AAV) vector transduced the inner retina with high efficiency in a rodent model. In large animals, however, the efficiency of retinal transduction was low, because the vitreous and internal limiting membrane (ILM) acted as barriers to transduction. To overcome these barriers in cynomolgus monkeys, we performed vitrectomy (VIT) and ILM peeling before AAV vector injection. Following intravitreal injection of 50 μL triple-mutated self-complementary AAV serotype 2 vector encoding EGFP, transduction efficiency was analyzed. Little expression of GFP was detected in the control and VIT groups, but in the VIT+ILM group, strong GFP expression was detected within the peeled ILM area. To detect potential adverse effects, we monitored the retinas using color fundus photography, optical coherence tomography, and electroretinography. No serious side effects associated with the pretreatment were observed. These results indicate that surgical ILM peeling before AAV vector administration would be safe and useful for efficient transduction of the nonhuman primate retina and provide therapeutic benefits for the treatment of retinal diseases.
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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.