Gene Therapy & The Eye

    Today only the symptoms of genetic retinal diseases like FEVR, Macular Degeneration (MD) or  Retinitis Pigmentosa (RP) can be treated.  While much improved technology such as lasers and fiber-optics have become vision saving technologies, an actual cure of a genetic diseases must lie in gene therapy.  While the evolution of gene therapy research has been a bumpy road recent success stories in animal research has paved a new road to human gene therapy research.  Most notably is the curing of MD and RP in dogs! (see articles below)

    Each one of us came into this world with genes inherited from our parents.  Sometimes one or more of these genes is defective.  A copy of our genes are located in each one of our cells in our bodies.  Gene therapy is the introduction and incorporation of a new, correct version, of a gene sequence into a body.  The first step before gene therapy can be preformed is to identify and understand what the defective gene is and is doing to our bodies.  The next step is to clone the gene and develop a genetic test.  The last step is to find a way to introduce the new genes to the body. 

    FEVR is actually a description of a set of problems with the retina and the vitreous humor of the eye.  This set of problems can be caused by different bad genes.   Three different inheritances, dominant, x-linked and recessive, are known to cause FEVR. 

Information on the inheritance of FEVR.

Autosomal dominant (AD) is the most common form of inheritance of FEVR. The frizzled-4 gene was recently discovered in 11q chromosome as one of the genes that cause AD FEVR. Another locus for AD disease is in 11p, the gene is still unknown. It is quite possible that few other unknown yet genes might cause AD FEVR.

The second most common form of tranmsmission is x-linked. Patients with the x-linked form have quite often mutations in the Norrie disease gene. It is quite possible also that this type of FEVR could also be caused by other genes as well.

A third type of inheritance is autosomal recessive, it is the least common and genes are still unknown.

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Articles On Curing Retinal Diseases with Gene Therapy

Will gene therapy that restored sight in dogs born blind prove successful in human clinical trials?

 

Future Gene Therapy Possible for Inherited Macular Degeneration Patterns

 

 GENE THERAPY MAY BE A TOOL TO PREVENT BLINDNESS - "Gene therapy may one day be used to halt or even prevent the overgrowth of blood vessels in the eye .... "

 

Advances in Gene Therapy - ... A similar experiment using AAV cured dogs of ... retinitis pigmentosa."