UPenn using Silence and Replace too

A US led collab looking to clinical trials for retinitis pigmentosa using same strategy as benitec's OPMD - shrna silence and gene replace via same vector, following success with dogs in pre-clinical work.

“We were able to save cone vision in the dogs,” says Florida’s Lewin. “If we can do that in people, it will save the central vision that allows them to recognize faces, read and watch television.”

Thus far, tracking the treatment effect more than eight months after delivery of the gene therapy, the effect seems stable and lasting. The research team is currently working to move the findings into clinical trials.