Green fluorescent protein in photoreceptors and retinal pigment epithelial cells after subretinal injection of AAV8 in a monkey retina. Cell nuclei are labeled blue and cone photoreceptors are labeled red.

It is now 21 years since the first human gene therapy clinical trial, the field is still in “pioneering” mode to find the right combination of gene, cell and delivery method for therapeutic benefit.  Recently, clinical investigators at the University of Pennsylvania used an adeno-associated virus to deliver a gene to retinal cells of children suffering from Leber’s congenital amaurosis and were able to restore light reception.  The future for this approach to other eye diseases rests on controlling gene expression and finding the most efficient viral vectors to target tissue types. MORE

Image Credit: Luk Vandenberghe, Ph.D., Perelman School of Medicine at the University of Pennsylvania and Eurekaalert.org