ArticlePeerJ2019
Evaluation of AAV-DJ vector for retinal gene therapy.
Article in PeerJ, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
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Who cites it
27 citing papers in PubMed, 47 citations in OpenAlex.
- Photobiology in ophthalmology: from evolutionary adaptation to translational therapeutics.Japanese journal of ophthalmology · 2026Review
- AAV-based gene therapies for neovascular AMD.Gene therapy · 2026Review
- Tropism and Retinal Transduction Efficiency of Adeno-Associated Virus Serotypes in Mice.Investigative ophthalmology & visual science · 2025Article
- Tropism of the AAV6.2 Vector in the Murine Retina.International journal of molecular sciences · 2025Article
- Ocular toxicity, distribution, and shedding of intravitreal AAV-eqIL-10 in horses.Molecular therapy. Methods & clinical development · 2024Article
- Challenges in AAV-Based Retinal Gene Therapies and the Role of Magnetic Nanoparticle Platforms.Journal of clinical medicine · 2024Review
- Gene Therapy in the Light of Lifestyle Diseases: Budesonide, Acetaminophen and Simvastatin Modulates rAAV Transduction Efficiency.Pharmaceuticals (Basel, Switzerland) · 2024Article
- Transduction and Genome Editing of the Heart with Adeno-Associated Viral Vectors Loaded onto Electrospun Polydioxanone Nonwoven Fabrics.Biomolecules · 2024Article
- Intravitreal injection of new adeno-associated viral vector: Enhancing retinoschisin 1 gene transduction in a mouse model of X-linked retinoschisis.Biochemistry and biophysics reports · 2024Article
- In utero adeno-associated virus (AAV)-mediated gene delivery targeting sensory and supporting cells in the embryonic mouse inner ear.PloS one · 2024Article
- Highly sensitive visual restoration and protection via ectopic expression of chimeric rhodopsin in mice.iScience · 2023Article
- Starburst amacrine cells amplify optogenetic visual restoration through gap junctions.Molecular therapy. Methods & clinical development · 2023Article
- Article
- Improved gene therapy for MFRP deficiency-mediated retinal degeneration by knocking down endogenous bicistronicMolecular therapy. Nucleic acids · 2023Article
- Retinitis Pigmentosa: Novel Therapeutic Targets and Drug Development.Pharmaceutics · 2023Review
- Adeno-associated virus vectors for retinal gene therapy in basic research and clinical studies.Frontiers in medicine · 2023Review
- Intravitreal Injection of AAV for the Transduction of Mouse Retinal Ganglion Cells.Methods in molecular biology (Clifton, N.J.) · 2023Article
- AAV2 and AAV9 tropism and transgene expression in the mouse eye and major tissues after intravitreal and subretinal delivery.Frontiers in drug delivery · 2023Article
- AAV vectors: The Rubik's cube of human gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Review
- Intracameral Injection of AAV-DJ.COMP-ANG1 Reduces the IOP of Mice by Reshaping the Trabecular Outflow Pathway.Investigative ophthalmology & visual science · 2022Article
Corrections and comments
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Authors and funding
4 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeThe most common virus vector used in gene therapy research for ophthalmologic diseases is the adeno-associated virus (AAV) vector, which has been used successfully in a number of preclinical and clinical studies. It is important to evaluate novel AAV vectors in animal models for application of clinical gene therapy. The AAV-DJ (type 2/type 8/type 9 chimera) was engineered from shuffling eight different wild-type native viruses. In this study, we investigated the efficiency of gene transfer by AAV-DJ injections into the retina.
methodsOne microliter of AAV-2-CAGGS-EGFP or AAV-DJ-CAGGS-EGFP vector at a titer of 1.4 × 10e12 vg/ml was injected intravitreally or subretinally in each eye of C57BL/6 mice. We evaluated the transduction characteristics of AAV-2 and -DJ vectors using fluorescence microscopy and electroretinography.
resultsThe results confirmed that AAV-DJ could deeply transfer gene to photoreceptor layer with intravitreal injection and has an efficient gene transfer to various cell types especially the Mueller cells in the retina. Retinal function was not affected by AAV-DJ infection or ectopic EGFP expression.
conclusionsThe AAV-DJ vector efficiently induces the reporter gene in both the inner and outer murine retina without functional toxicity. These data indicated that the AAV-DJ vector is a useful tool for the gene therapy research targeting retinal disorders.
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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.