ArticleInvestigative ophthalmology & visual science2013
Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: safety and biodistribution of StarGen for Stargardt disease.
Article in Investigative ophthalmology & visual science, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers.
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Who cites it
62 citing papers in PubMed, 108 citations in OpenAlex.
- Three-Year Safety Results of SAR422459 (EIAV-ABCA4) Gene Therapy in Patients With ABCA4-Associated Stargardt Disease: An Open-Label Dose-Escalation Phase I/IIa Clinical Trial, Cohorts 1-5.American journal of ophthalmology · 2022Trial
- Update on the Management of ABCA4 Retinopathy (Stargardt Disease).Ophthalmology and therapy · 2026Review
- Comprehensive multiomics analyses identify guanylate cyclase activator 1A as a photoreceptor-specific target in a murine model of oxygen-induced retinopathy.Journal of translational medicine · 2026Article
- Systematic empirical evaluation of individual base editing targets: Validating therapeutic targets in USH2A and comparison of methods.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Emerging Therapeutic Approaches and Genetic Insights in Stargardt Disease: A Comprehensive Review.International journal of molecular sciences · 2024Review
- Recurrent and Concurrent Prediction of Longitudinal Progression of Stargardt Atrophy and Geographic Atrophy.medRxiv : the preprint server for health sciences · 2024Article
- How Advanced are Nanocarriers for Effective Subretinal Injection?International journal of nanomedicine · 2024Review
- Losing, preserving, and restoring vision from neurodegeneration in the eye.Current biology : CB · 2023Review
- Lentiviral delivered aflibercept OXB-203 for treatment of neovascular AMD.Molecular therapy. Methods & clinical development · 2023Article
- Using Ensemble OCT-Derived Features beyond Intensity Features for Enhanced Stargardt Atrophy Prediction with Deep Learning.Applied sciences (Basel, Switzerland) · 2023Article
- How to Set Up Genetic Counselling for Inherited Macular Dystrophies: Focus on Genetic Characterization.International journal of molecular sciences · 2023Review
- Artificial intelligence for assessment of Stargardt macular atrophy.Neural regeneration research · 2022Review
- Nanomedicine and drug delivery to the retina: current status and implications for gene therapy.Naunyn-Schmiedeberg's archives of pharmacology · 2022Review
- Effective gene therapy of Stargardt disease with PEG-ECO/Molecular therapy. Nucleic acids · 2022Article
- Update on Viral Gene Therapy Clinical Trials for Retinal Diseases.Human gene therapy · 2022Review
- Lentiviral Vectors for Ocular Gene Therapy.Pharmaceutics · 2022Review
- Review
- Current trends in gene therapy for retinal diseases (Review).Experimental and therapeutic medicine · 2022Review
- Ocular Gene Therapy: A Literature Review with Special Focus on Immune and Inflammatory Responses.Clinical ophthalmology (Auckland, N.Z.) · 2022Review
- An Overview of the Genetics ofGenes · 2021Review
2 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
25 authors at 3 institutions in 2 countries.
Funding
Abstract
purposeStarGen is an equine infectious anemia virus (EIAV)-based lentiviral vector that expresses the photoreceptor-specific adenosine triphosphate (ATP)-binding cassette transporter (ABCA4) protein that is mutated in Stargardt disease (STGD1), a juvenile macular dystrophy. EIAV vectors are able to efficiently transduce rod and cone photoreceptors in addition to retinal pigment epithelium in the adult macaque and rabbit retina following subretinal delivery. The safety and biodistribution of StarGen following subretinal delivery in macaques and rabbits was assessed.
methodsRegular ophthalmic examinations, IOP measurements, ERG responses, and histopathology were carried out in both species to compare control and vector-treated eyes. Tissue and fluid samples were obtained to evaluate the persistence, biodistribution, and shedding of the vector following subretinal delivery.
resultsOphthalmic examinations revealed a slightly higher level of inflammation in StarGen compared with control treated eyes in both species. However, inflammation was transient and no overt toxicity was observed in StarGen treated eyes and there were no abnormal clinical findings. There was no StarGen-associated rise in IOP or abnormal ERG response in either rabbits or macaques. Histopathologic examination of the eyes did not reveal any detrimental changes resulting from subretinal administration of StarGen. Although antibodies to StarGen vector components were detected in rabbit but not macaque serum, this immunologic response did not result in any long-term toxicity. Biodistribution analysis demonstrated that the StarGen vector was restricted to the ocular compartment.
conclusionsIn summary, these studies demonstrate StarGen to be well tolerated and localized following subretinal administration.
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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.