ArticleGene therapy2023
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters.
Article in Gene therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 49 papers.
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Who cites it
49 citing papers in PubMed, 72 citations in OpenAlex.
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- Streamlined synthetic regulatory cassette for efficient and photoreceptor-enriched retinal gene expression.Molecular therapy. Nucleic acids · 2026Article
- Culture media alter retinal organoid physiology promoting AAV transduction and retinal ganglion cell survival.Gene therapy · 2026Article
- AAV-based gene therapies for neovascular AMD.Gene therapy · 2026Review
- Neuritin1 Cis-Regulatory Elements Enable Gene Expression Preferentially in Retinal Ganglion Cells.Investigative ophthalmology & visual science · 2026Article
- Combination gene therapy with AAV based RPE65 and survivin vectors sustains phenotypic rescue in neural retina of LCA2 mice.Molecular and cellular biochemistry · 2026Article
- Identification of Recombinant Adeno-Associated Virus Serotypes by Matrix-Assisted Laser Desorption/Ionization Mass Spectrometry.Analytical chemistry · 2026Article
- Establishment of a Sensitized 3D Spheroid Cancer Cell Model for Enhanced Anti-Cancer Drug Discovery.Methods and protocols · 2026Article
- Identification a Compact Promoter using a New Promoter Selection Strategy and Engineering Hybrid Pol II/III Enable Efficient Genome Editing in Human Retinal Ganglion Cells.bioRxiv : the preprint server for biology · 2026Article
- Designing synthetic regulatory elements using the generative AI framework DNA-Diffusion.Nature genetics · 2026Article
- Balancing Promise and Peril: Hemophilia Gene Therapy Insights.IUBMB life · 2026Review
- Preclinical evaluation of AL-001, a gene therapy for wet age-related macular degeneration.Frontiers in genetics · 2026Article
- Characterization of adeno-associated viral transduction of retinal ganglion cells in adult and old mice.Molecular therapy. Methods & clinical development · 2025Article
- In vivo mitochondrial base editing restores genotype and visual function in a mouse model of LHON.Nature communications · 2025Article
- In vivo validation of novel non-invasive PHP.eB AAVs as a potential therapeutic approach for alpha-synucleinopathies.Acta neuropathologica communications · 2025Article
- Simultaneous isolation and parallel analysis of genomic DNA and total RNA: A comprehensive approach.Molecular therapy. Methods & clinical development · 2025Article
- Potentiation of mitochondrial function by mitoDREADD-GNature neuroscience · 2025Article
- Large-scale survey, animal models, and computational modeling identify histological neurodegenerative biomarkers for traumatic optic neuropathy.JCI insight · 2025Article
- Optogenetic restoration of high-sensitivity vision using ChRmine- and ChroME-based channelrhodopsins.Scientific reports · 2025Article
- Targeted spiral ganglion neuron degeneration in parvalbumin-Cre neonatal mice.Molecular therapy. Methods & clinical development · 2025Article
Corrections and comments
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Authors and funding
8 authors at 4 institutions in 6 countries.
Funding
Abstract
Recombinant adeno-associated viral vectors (AAVs) are an effective system for gene transfer. AAV serotype 2 (AAV2) is commonly used to deliver transgenes to retinal ganglion cells (RGCs) via intravitreal injection. The AAV serotype however is not the only factor contributing to the effectiveness of gene therapies. Promoters influence the strength and cell-selectivity of transgene expression. This study compares five promoters designed to maximise AAV2 cargo space for gene delivery: chicken β-actin (CBA), cytomegalovirus (CMV), short CMV early enhancer/chicken β-actin/short β-globulin intron (sCAG), mouse phosphoglycerate kinase (PGK), and human synapsin (SYN). The promoters driving enhanced green fluorescent protein (eGFP) were examined in adult C57BL/6J mice eyes and tissues of the visual system. eGFP expression was strongest in the retina, optic nerves and brain when driven by the sCAG and SYN promoters. CBA, CMV, and PGK had moderate expression by comparison. The SYN promoter had almost exclusive transgene expression in RGCs. The PGK promoter had predominant expression in both RGCs and AII amacrine cells. The ubiquitous CBA, CMV, and sCAG promoters expressed eGFP in a variety of cell types across multiple retinal layers including Müller glia and astrocytes. We also found that these promoters could transduce human retina ex vivo, although expression was predominantly in glial cells due to low RGC viability. Taken together, this promoter comparison study contributes to optimising AAV-mediated transduction in the retina, and could be valuable for research in ocular disorders, particularly those with large or complex genetic cargos.
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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.