ArticlePloS one2011
Retinal degeneration progression changes lentiviral vector cell targeting in the retina.
Article in PloS one, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 28 citations in OpenAlex.
- Novel photoreceptor-specific promoters for gene therapy in mid- to late-stage retinal degeneration.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- A Long-Term Safety and Efficacy Report on Intravitreal Delivery of Adipose Stem Cells and Secretome on Visual Deficits After Traumatic Brain Injury.Translational vision science & technology · 2022Article
- Lentiviral Vectors for Ocular Gene Therapy.Pharmaceutics · 2022Review
- Helper-Dependent Adenovirus Transduces the Human and Rat Retina but Elicits an Inflammatory Reaction When Delivered Subretinally in Rats.Human gene therapy · 2019Article
- Expression of ABCA4 in the retinal pigment epithelium and its implications for Stargardt macular degeneration.Proceedings of the National Academy of Sciences of the United States of America · 2018Article
- Rod Outer Segment Development Influences AAV-Mediated Photoreceptor Transduction After Subretinal Injection.Human gene therapy · 2017Article
- Gene therapy using stem cells.Cold Spring Harbor perspectives in medicine · 2014Review
- Vector platforms for gene therapy of inherited retinopathies.Progress in retinal and eye research · 2014Review
- Retinal transduction profiles by high-capacity viral vectors.Gene therapy · 2014Article
- Drug and gene delivery to the back of the eye: from bench to bedside.Investigative ophthalmology & visual science · 2014Review
- Targeting neurons and photons for optogenetics.Nature neuroscience · 2013Review
- Loss of Otx2 in the adult retina disrupts retinal pigment epithelium function, causing photoreceptor degeneration.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2013Article
- Gene therapy for retinal disease.Translational research : the journal of laboratory and clinical medicine · 2013Review
- Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients.Human gene therapy · 2012Review
- Advances in repairing the degenerate retina by rod photoreceptor transplantation.Biotechnology advancesReview
Corrections and comments
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Authors and funding
9 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In normal mice, the lentiviral vector (LV) is very efficient to target the RPE cells, but transduces retinal neurons well only during development. In the present study, the tropism of LV has been investigated in the degenerating retina of mice, knowing that the retina structure changes during degeneration. We postulated that the viral transduction would be increased by the alteration of the outer limiting membrane (OLM). Two different LV pseudotypes were tested using the VSVG and the Mokola envelopes, as well as two animal models of retinal degeneration: light-damaged Balb-C and Rhodopsin knockout (Rho-/-) mice. After light damage, the OLM is altered and no significant increase of the number of transduced photoreceptors can be obtained with a LV-VSVG-Rhop-GFP vector. In the Rho-/- mice, an alteration of the OLM was also observed, but the possibility of transducing photoreceptors was decreased, probably by ongoing gliosis. The use of a ubiquitous promoter allows better photoreceptor transduction, suggesting that photoreceptor-specific promoter activity changes during late stages of photoreceptor degeneration. However, the number of targeted photoreceptors remains low. In contrast, LV pseudotyped with the Mokola envelope allows a wide dispersion of the vector into the retina (corresponding to the injection bleb) with preferential targeting of Müller cells, a situation which does not occur in the wild-type retina. Mokola-pseudotyped lentiviral vectors may serve to engineer these glial cells to deliver secreted therapeutic factors to a diseased area of the retina.
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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.