ArticleVirus research2020
Exosome-associated SUMOylation mutant AAV demonstrates improved ocular gene transfer efficiency in vivo.
Article in Virus research, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 1 of them a synthesis that pooled it.
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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.
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Who cites it
13 citing papers in PubMed, 1 synthesis or guideline pooled it, 22 citations in OpenAlex.
- Harnessing exosomes in dry eye disease: a triple threat approach.BMC ophthalmology · 2026Pooled it
- Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Scientometric analysis of extracellular vesicles in vision science (up to 2024).Journal of nanobiotechnology · 2025Review
- Exploring AAV-Mediated Gene Therapy for Inner Ear Diseases: from Preclinical Success to Clinical Potential.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- Small molecule inhibition of SUMOylation increases expression from AAV vectors both during and after initial transduction in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- The role of post-translational modifications in parvovirus life cycle.Frontiers in veterinary science · 2025Review
- Challenges in AAV-Based Retinal Gene Therapies and the Role of Magnetic Nanoparticle Platforms.Journal of clinical medicine · 2024Review
- Development of an Optimized Promoter System for Exosomal and Naked AAV Vector-Based Suicide Gene Therapy in Hepatocellular Carcinoma.ACS omega · 2024Article
- Targeting the Ophthalmic Diseases Using Extracellular Vesicles 'Exosomes': Current Insights on Their Clinical Approval and Present Trials.Aging and disease · 2024Review
- Extracellular Vesicles as Novel Drug-Delivery Systems through Intracellular Communications.Membranes · 2022Review
- Neutralizing Antibody Evasion and Transduction with Purified Extracellular Vesicle-Enveloped Adeno-Associated Virus Vectors.Human gene therapy · 2021Article
- Impact of Medium-Sized Extracellular Vesicles on the Transduction Efficiency of Adeno-Associated Viruses in Neuronal and Primary Astrocyte Cell Cultures.International journal of molecular sciences · 2021Article
- Exosome-Shuttled circSHOC2 from IPASs Regulates Neuronal Autophagy and Ameliorates Ischemic Brain Injury via the miR-7670-3p/SIRT1 Axis.Molecular therapy. Nucleic acids · 2020Article
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
Exosome associated Adeno-associated virus (AAV) vectors have emerged as a promising tool in gene therapy. Recently, we elucidated the role of SUMOylation post-translational modification in AAV2 capsid and demonstrated that capsid modifications at SUMOylation sites, enhance vector transduction. The present study was designed to study the combinatorial effect of exosome delivery of a SUMOylation site modified AAV2, during ocular gene therapy. In the first set of experiments, we investigated the in vitro gene transfer potential of exosome-associated SUMOylation mutant AAV2 (Exo-K105Q-EGFP) in human retinal pigmental epithelial (ARPE19) cells. Our data showed that, Exo-K105Q vectors had a significantly higher transduction potential in ARPE19 cells when compared to exosomes derived from wildtype AAV2 (Exo-AAV2-EGFP) vector packaging. Subsequently, an intravitreal administration of exosome associated mutant AAV2 vectors in C57BL6/J mice, demonstrated a significant increase reporter gene (EFGP) expression 4 weeks after gene transfer. Further immunostaining, revealed that these exosome-based vectors also had a better permeation across the retinal layers. These data highlight the translational potential of exosome associated SUMOylation mutant AAV for ocular gene therapy.
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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.