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Thursday, September 17, 2009

Gene therapy for colour blindness

Researchers have used gene therapy to restore colour vision in two adult monkeys that have been unable to distinguish between red and green hues since birth — raising the hope of curing colour blindness and other visual disorders in humans.

About 1 in 12 men lack either the red- or the green-sensitive photoreceptor proteins that are normally present in the colour-sensing cells or cones of the retina and so have red–green colour blindness. If we can target gene expression specifically to cones, in humans, then this has a tremendous implication.

Three human gene therapy trials are currently under way for loss of sight due to serious degeneration of the retina. These phase I safety studies injected a similar type of virus vector (but carrying a different gene) behind the retina as in the monkeys, and people treated have shown no serious adverse effects more than a year after, with some participants reporting marked improvements in vision

References

(1) Mancuso, K.
et al. Nature advanced online publication, doi:10.1038/nature08401 (2009).

(2) Cideciyan, A. V.
et al. N. Engl. J. Med. 361, 725-727 (2009).

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