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Updated: May 31, 2026
06:48
Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
Published on: August 7, 2015
Republished review: Gene therapy for ocular diseases
Melissa M Liu1, Jingsheng Tuo, Chi-Chao Chan
1Immunopathology Section, Laboratory of Immunology, National Eye Institute, National Institutes of Health, 10 Center Drive, Bldg 10, Rm 10N103, NIH/NEI, Bethesda, MD 20895-1857, USA.
Postgraduate Medical Journal
|June 28, 2011
Summary
Ocular gene therapy shows promise for treating genetic eye diseases. Early clinical trials for Leber congenital amaurosis are reporting preliminary successes, advancing treatment for various retinal and non-retinal conditions.
Area of Science:
- Ophthalmology
- Genetics
- Molecular Biology
Background:
- The eye's accessibility and immune privilege make it an ideal target for gene therapy.
- Genetic basis of ocular diseases is increasingly understood, paving the way for gene replacement and silencing therapies.
- Vector-based gene transfer methods have seen improvements in safety and specificity.
Purpose of the Study:
- To review current developments in ocular gene therapy.
- To discuss future prospects for treating genetic eye diseases.
- To highlight advancements in vector-mediated gene transfer for ocular conditions.
Main Methods:
- Review of current literature on ocular gene therapy research.
- Analysis of advancements in recombinant adeno-associated virus (AAV) and lentivirus vectors.
- Examination of experimental models for ocular diseases.
Main Results:
- Phase 1 clinical trials for Leber congenital amaurosis show preliminary success.
- Gene therapy approaches are advancing for retinitis pigmentosa, retinoschisis, Stargardt disease, and age-related macular degeneration.
- Experimental models demonstrate progress for non-retinal diseases like uveitis and glaucoma.
Conclusions:
- Ocular gene therapy is progressing rapidly, with early clinical successes reported.
- Novel vector technologies are enhancing gene transfer efficiency and regulation.
- Methodological advancements are crucial for future gene-based therapies for human eye diseases.
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