Envisioning Gene Transfer: Report on Congenital Blindness Studies

This week, the New England Journal of Medicine published two preliminary trial reports using gene transfer against a rare form of congenital blindness– Leber's Congenital Amaurosis type 2.  Since Jean Bennett and coworkers at the University of Pennsylvania reported in 2001 successfully correcting visual deficiencies in dogs afflicted with a nearly identical genetic condition, the field of gene transfer has been atwitter with anticipation of these results.

In the next few posts, I will comment on the studies, the science, the ethics, and the reporting of the LCA studies. (photo credit: chrisphoto, optomap retina scan, 2005)