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Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors
K S Balaggan1, K Binley, M Esapa
1Division of Molecular Therapy, Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
The Journal of Gene Medicine
|November 22, 2005
Summary
Minimal equine infectious anaemia virus (EIAV) vectors show potential for ocular gene therapy. These vectors efficiently transduced retinal cells and corneal cells, offering a promising tool for treating eye disorders.
Area of Science:
- Ophthalmology
- Gene Therapy
- Virology
Background:
- Development of minimal non-primate lentiviral vectors based on equine infectious anaemia virus (EIAV).
- Evaluation of in vivo expression profiles of EIAV vectors for ocular gene therapy applications.
Purpose of the Study:
- To define the potential utility of EIAV vectors in ocular gene therapy.
- To analyze the in vivo expression profiles of EIAV vectors delivered to ocular tissues.
Main Methods:
- EIAV vectors pseudotyped with VSV-G or rabies-G were administered via subretinal, intravitreal, or intracameral routes in mice.
- Reporter gene (eGFP) expression was assessed using in vivo imaging and histology up to 16 months.
- Factors influencing expression, including vector titre, pseudotype, administration site, and retinal trauma, were investigated.
Main Results:
- Subretinal delivery led to stable transduction of retinal pigment epithelial (RPE) cells and photoreceptors for up to 16 months.
- Retinal trauma enhanced transduction of neurosensory retinal cells.
- Intracameral delivery of VSV-G pseudotyped vectors resulted in stable expression in corneal endothelial and trabecular meshwork cells.
Conclusions:
- Optimized EIAV vectors demonstrate suitable cellular tropism and expression kinetics for intraocular gene delivery.
- These vectors are promising for treating inherited and acquired retinal and anterior segment disorders.
- EIAV vectors offer a viable alternative for ocular gene therapy strategies.