Since news that researchers had restored sight in dogs with Leber congenital amaurosis (LCA) broke two weeks ago, Jean Bennett's phones at the F.M. Kirby Center for Molecular Ophthalmology at University of Pennsylvania's Scheie Eye Institute haven't stopped ringing. Anxious parents, whose infants suffer from this and other retinal degenerative diseases, want help. But they have to wait a few years; much more needs to be done. Researchers at Penn, Cornell University, and the University of Florida delivered a good copy of the gene defective in LCA, RPE65, to the right eyes of afflicted dogs using adeno-associated virus. Success was measured qualitatively with numerous response experiments, quantitatively using electroretinography, and molecularly using PCR and western blot analysis (G. Acland et al., "Gene therapy restores vision in a canine model of childhood blindness," Nature Genetics, 28:92-5, May 2001). Now, 10 months later, sight in the treated dogs' right...
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