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Abstract
Gene therapy is the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state .It is a new form of drug delivery that could potentially eliminate the need for repeated administration of proteins or drugs. Applications of gene therapy not only include rare inherited diseases but extend to common acquired disorders. there are two main types of gene therapy , somatic cell gene therapy which involves gene transfer to body cells other than those involved in reproduction , this can be done Ex-vivo or In-vivo , and germ-line gene therapy which is the ex vivo genetic modification of gametes or pre-implantation embryos .
The eye is one of the most suitable targets for gene therapy. It is easily accessible and allows local application of therapeutic agents with reduced risk of systemic effects. In addition, the effects of treatment may be monitored by a variety of non-invasive examinations. In the eye, the retina is possibly the best candidate for gene therapy. The amount of virus injected into the retina is about 1/1000 of the amount used for systemic diseases. Moreover, there are many established small and large animal models of retinal disease. Retinal diseases, Optic neuritis, retinoblastoma ,CNV, PVR , glaucoma , uveitis , corneal diseases and PCO are ocular diseases where gene therapy has been exploited with success .
In order to provide a benefit in the treatment of a disease, a gene-drug must be transferred to the desired tissue .Vectors are the vehicles that are used for gene transfer .There are two types of delivery systems: viral and non viral . Retroviral vectors have high efficiency of gene transfer into replicating cells. they have great potential in disorders associated with neovascularization .