ArticleMolecular therapy : the journal of the American Society of Gene Therapy2009
Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 202 papers, 3 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
202 citing papers in PubMed, 3 syntheses or guidelines pooled it, 345 citations in OpenAlex.
- Sharper vision, steady hands: can robots improve subretinal drug delivery? Systematic review.Journal of robotic surgery · 2024Pooled it
- A Systematic Review of Optogenetic Vision Restoration: History, Challenges, and New Inventions from Bench to Bedside.Cold Spring Harbor perspectives in medicine · 2023Pooled 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
- Developmental synchrony of retinal waves, apoptosis, and angiogenesis in postnatal retina.eLife · 2026Article
- Deep-sequencing-guided library screening and profiling of AAV capsids in the primate retina.Molecular therapy. Advances · 2026Article
- Optimization of AAV tools to target Müller glial cells for retinal gene therapy.Molecular therapy. Advances · 2026Article
- Lipid nanoparticles enable mRNA delivery to diverse cell types of the inner Retina.Molecular therapy. Nucleic acids · 2026Article
- Phase I/IIa Study of an Intravitreal Optogenetic Therapy (AGN-151597) in Patients with Advanced Retinitis Pigmentosa.Ophthalmology science · 2026Article
- A low-cost, tunable suprachoroidal injection platform with real-time OCT guidance for gene therapy delivery.Experimental eye research · 2026Article
- AAV Vectors in Regenerative Medicine and Cellular Reprogramming: Potential, Pitfalls, and Specificity Constraints.International journal of molecular sciences · 2026Review
- Strategic lipid nanoparticle design dictates retinal delivery post inner limiting membrane disruption.Drug delivery and translational research · 2026Article
- The internal limiting basement membrane inhibits functional engraftment of transplanted human retinal ganglion cells in vivo.Science translational medicine · 2026Article
- Advancing nanoparticle delivery studies: flow cytometry-based quantification of nanoparticle uptake and transfection in retinal tissue.Drug delivery and translational research · 2026Article
- AAV2-mediated intravitreal delivery of exon-specific U1 snRNA rescues optic neuropathy in familial dysautonomia.Molecular therapy. Nucleic acids · 2026Article
- Interrogating the dependency of AAV capsids on AAVR for retinal transduction.Molecular therapy. Advances · 2026Article
- AAV2-Retro-Mediated Gene Transfer Selectively Targets Outer Retinal Cells Following Intravitreal Injection.Investigative ophthalmology & visual science · 2026Article
- Review
- Anatomically Guided Non-Viral CRISPR/Cas9 Delivery in the Eye: Overcoming Barriers for Precision Gene Therapy.Pharmaceutics · 2026Review
- 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
142 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 1 institution in 1 country.
Funding
Abstract
Adeno-associated viral gene therapy has shown great promise in treating retinal disorders, with three promising clinical trials in progress. Numerous adeno-associated virus (AAV) serotypes can infect various cells of the retina when administered subretinally, but the retinal detachment accompanying this injection induces changes that negatively impact the microenvironment and survival of retinal neurons. Intravitreal administration could circumvent this problem, but only AAV2 can infect retinal cells from the vitreous, and transduction is limited to the inner retina. We therefore sought to investigate and reduce barriers to transduction from the vitreous. We fluorescently labeled several AAV serotype capsids and followed their retinal distribution after intravitreal injection. AAV2, 8, and 9 accumulate at the vitreoretinal junction. AAV1 and 5 show no accumulation, indicating a lack of appropriate receptors at the inner limiting membrane (ILM). Importantly, mild digestion of the ILM with a nonspecific protease enabled substantially enhanced transduction of multiple retinal cell types from the vitreous, with AAV5 mediating particularly remarkable expression in all retinal layers. This protease treatment has no effect on retinal function as shown by electroretinogram (ERG) and visual cortex cell population responses. These findings may help avoid limitations, risks, and damage associated with subretinal injections currently necessary for clinical gene therapy.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
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.