Sunday, August 11, 2013

How the therapy works

I'm going to try and explain what the trial will do, as simply and in as few words as I can.

The researchers have created a functioning RPE65 gene. They insert this functioning gene into a virus, known as a vector.

The vector is then injected  underneath the retina in the back of the eye. The virus releases the normal gene into the eye, where it begins to replicate and manufacture the protein necessary for normal retinal function.

As long as the retinal cells have not scarred over completely, they can start to function again. This can takes days to weeks to months.

But the theory is, the younger the patient, the less scarring and the more vision that can be restored.


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