Let There Be Light

Science Signaling  27 Jul 2010:
Vol. 3, Issue 132, pp. ec232
DOI: 10.1126/scisignal.3132ec232

Retinitis pigmentosa, a disease that can result from a wide variety of genetic defects, causes degeneration of photoreceptor cells in the retina and leads to blindness. In the course of the disease, it is generally the rod photoreceptor cells that degenerate first. Cone photoreceptor cells may persist, but in a damaged and nonfunctional state. Busskamp et al. (see the Perspective by Cepko) have now applied a gene therapy approach to mouse models of retinitis pigmentosa. Inducing expression of a bacterial light-activated ion pump, halorhodopsin, in the damaged cone cells improved visual responses in the diseased mouse retinas. Thus, it may be possible to rescue cone photoreceptors therapeutically, even after they have already been damaged.

V. Busskamp, J. Duebel, D. Balya, M. Fradot, T. J. Viney, S. Siegert, A. C. Groner, E. Cabuy, V. Forster, M. Seeliger, M. Biel, P. Humphries, M. Paques, S. Mohand-Said, D. Trono, K. Deisseroth, J. A. Sahel, S. Picaud, B. Roska, Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa. Science 329, 413–417 (2010). [Abstract] [Full Text]

C. Cepko, Seeing the light of day. Science 329, 403–404 (2010). [Summary] [Full Text]