In Vivo Gene Transfer to the Mouse Eye Using an HIV-Based Lentiviral Vector; Efficient Long-Term Transduction of Corneal Endothelium and Retinal Pigment Epithelium
Abstract
We have evaluated the transduction profiles of an HIV-based lentiviral vector delivered regionally to ocular tissues in vivo. Following subretinal injection, a green fluorescent protein (GFP) reporter gene was efficiently and stably expressed in retinal pigment epithelial (RPE) cells. Limited transduction of adjacent photoreceptors occurred in newborn mice, but was inefficient in adult animals. Injection of the vector into the anterior chamber resulted in efficient and stable transduction of corneal endothelial cells. Efficient in vivo gene transfer into cells of the corneal endothelium and retinal pigment epithelium by lentiviral vectors may therefore offer a valuable approach to the treatment of disorders of the cornea and outer retina.
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In another study, HIV vectors carrying GFP delivered by subretinal injection showed stable expression in RPE and no inflammatory cell infiltration by immunostaining.
“In another study, HIV vectors carrying GFP were delivered by subretinal injection-a stable expression was observed in RPE, and no inflammatory cell infiltration was observed by immunostaining [57].”
From Immune responses to gene editing by viral and non-viral delivery vectors used in retinal gene therapy (2022) · cites this paper as [57]
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- Authors
- Bainbridge, J., Stephens, C., Parsley, K., Demaison, C., Halfyard, A., Thrasher, A., & Ali, R.
- Affiliations
- University College London (GB)
- Venue
- Gene Therapy
- Published
- 2001-11-01
- Volume
- 8
- Issue
- 21
- Pages
- 1665-1668
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- OpenAlex
- W1972583115
- Cited by
- 197
- References
- 13
- Citations by year
- 2026: 4 · 2025: 2 · 2024: 6 · 2023: 2 · 2022: 9 · 2021: 6 · 2020: 6 · 2019: 3 · 2018: 5 · 2017: 8 · 2016: 3 · 2015: 10 · 2014: 6 · 2013: 14 · 2012: 7
- FWCI
- 4.15
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- UCL Discovery (University College London) (submittedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinal Development and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- lentiviral vectors, HIV-based vectors, subretinal injection, anterior chamber injection, retinal pigment epithelium, corneal endothelium, photoreceptors, GFP expression, corneal disease, mouse eye
- MeSH terms
- Animals, Animals, Newborn, Eye, Eye Diseases, Genetic Vectors, Luminescent Proteins, Mice, Inbred C57BL, Microinjections, Pigment Epithelium of Eye, Transduction, Genetic, Genetic Therapy, HIV-1, Gene Expression, Epithelium, Corneal, Green Fluorescent Proteins, Mice
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