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Ophthalmology

Gene Therapy Retards Photoreceptor Degeneration in Retinitis Pigmentosa Model

Published in Gene Therapy Weekly, December 7th, 2000

In a rat model of retinitis pigmentosa, adeno-associated virus (AAV)-mediated gene transfer of basic fibroblast growth factor (FGF-2) was shown to preserve photoreceptors.

If the treatment can be applied to humans, it may be used to help slow progression of the disease, which can lead to blindness. Retinitis pigmentosa affects about one in every 4,000 people worldwide.

Optometrists at the University of California, Berkeley, injected subretinally in rats bovine FGF-2 gene via recombinant adeno-associated virus vector (rAAV) with a constitutive cytomegalovirus promoter. The transgenic rats (TgN S334ter-4) were 15 days old.

Eyes...

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