Intraocular delivery of recombinant virus.

In the past 5 yr, advances in technology have been made that allow efficient somatic transfer of functional genes to target cells in the eye in vivo. The ability to deliver functional genes to these cells is due primarily to the development of viral vectors-viruses in which various replicative functions have been replaced with transgene cassettes. Because progress continues in developing new vectors and refining those that are already available, the field has moved past the step of actually proving that gene transfer is possible to one where vectors are used (experimentally) for therapeutic purposes. In fact, proofs of principle of virus-based retinal gene therapy have been achieved in relevant animal models for retinitis pigmentosa (1-3).