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Abstract
The aim of this work is to study gene therapy in retinal dystrophies hoping that it will improve the prognosis of the diseases and the quality of life of the affected individuals.
that end Retinal dystrophies were some of eye threatining diseases with blindness. As they are all have genetic inheritance, the strategies of ophthalmology schools sighted upon gene therapy as promising goals.
A number of inherited retinal disorders are caused by mutation in genes expressed in the RPE as best disease or vitelliform macular dystrophy (VMD2), is an autosomal dominant disorder associated with macular visual loss in late adolescence.
Retinal dystrophies were some of eye threatining diseases that end with blindness. As they are all have genetic inheritance, the strategies of ophthalmology schools sighted upon gene therapy as promising goals.A number of inherited retinal disorders are caused by mutation in genes expressed in the RPE as best disease or vitelliform macular dystrophy (VMD2), is an autosomal dominant disorder associated with macular visual loss in late adolescence.Retinitis pigmentosa is a bilateral symmetrical, progressive disease whose onset is early in adult life. It start in the equatorial area of the retina and spreads centrally and peripherally but more rapidly in the later direction. The inheritance pattern may be autosomal dominant or recessive, x-linked, mitochondrial, or sporadic.Photoreceptor dystrophies are one of the commonest causes of inherited blindness in the western world. They may result from a defect in any one of over 50 different genes, many of which are photoreceptor specific There are many techniques to introduce the gene inside the retinal cells either by viral vector, electroporation, cationic lipid and polymers other molecular therapies include monoclonal antibodies, antisense oligonculeotides and ribozymes. On 01 May 2007 Moorfeilds Eye Hospital and university collage London’s institute of ophthalmology announced the world’s first gene therapy trial for inherited retinal dystrophy. The first operation was carried out on a 33 years old British male, Robert Johonson in early 2007 but it is currently too early for results.