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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
Published on: August 7, 2015
Potential applications for RNAi to probe pathogenesis and develop new treatments for ocular disorders
1Department of Ophthalmology, The Johns Hopkins University School of Medicine, Maumenee, Baltimore, MD 21287-9277, USA. pcampo@jhmi.edu
Gene Therapy
|October 1, 2005
Summary
Small interfering RNA (siRNA) shows promise for treating eye diseases like choroidal neovascularization and glaucoma. This RNA interference technology is being explored for new ocular therapies.
Area of Science:
- Ophthalmology
- Molecular Biology
- Genetics
Background:
- The eye's isolated nature makes it suitable for small interfering RNA (siRNA) therapies.
- Previous studies demonstrated siRNA's potential against choroidal neovascularization (CNV) by targeting VEGF or VEGFR1.
Purpose of the Study:
- To explore the utility of siRNA in understanding and treating various ocular diseases.
- To evaluate siRNA's role in conditions like CNV, corneal neovascularization, ocular scarring, glaucoma, and photoreceptor degeneration.
Main Methods:
- Utilizing siRNA targeting specific genes involved in ocular disease pathways, including VEGF, VEGFR1, and TGFbetaR2.
- Investigating siRNA's efficacy in both in vitro ocular cell cultures and in vivo animal models.
- Employing RNA interference (RNAi) to identify genes contributing to apoptosis and oxidative damage in retinal cells.
Main Results:
- VEGF and VEGFR1 siRNAs are effective against CNV and are in clinical trials.
- VEGFR1 siRNA confirms its pro-angiogenic role in the eye.
- Topical siRNA suppressed corneal neovascularization.
- TGFbetaR2 siRNA showed benefits in a model of ocular scarring post-glaucoma surgery.
- RNAi identified genes involved in retinal cell apoptosis and oxidative damage.
Conclusions:
- siRNA is a valuable tool for elucidating ocular disease pathogenesis.
- siRNA-based therapies hold significant potential for developing novel treatments for a range of eye conditions.
- Early in vivo and in vitro data support the therapeutic potential of siRNA in ophthalmology.
Related Concept Videos
01:23RNA Interference
RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
02:30siRNA - Small Interfering RNAs
Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...
02:15Experimental RNAi
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...