Howard Hughes Medical Institute, Laboratory of Molecular
Biology and Biochemistry, Rockefeller University, New York,
New York 10021, USA. sakmar@rockvax.rockefeller.edu
More than 100 genes have been mapped that cause inherited blinding
diseases. The majority affect the outer layer of the retina, which is composed
of photoreceptor cells and retinal pigment epithelium. Gene therapy is a potential
means of correcting defects in specialized cellular structures that mediate
phototransduction or visual pigment regeneration, as illustrated by a paper
in this issue.
