Big step forward in gene therapy technique for photoreceptors

We all know of the huge success obtained with gene therapy for RPE65 Leber’s Congenital Amaurosis, at least in dogs, which is paving the way for similar experiments with different LCA genes. There is however an important difference between RPE65 and most other genes that cause LCA. While the former is expressed in the retinal epithelium (RP), other LCA genes are expressed primarily in photoreceptors. Since photoreceptors are much more difficult to access than the RP, the methods used for the RPE65 trials may not work for photoreceptor-specific gene replacement. But this obstacle is being overcome.
Researchers led by Dr. Alberto Auricchio, of the Telethon Institute for Genetics and Medicine (TIGEM), based in Naples, Italy, has identified viral vectors that can access rods and cones of mice more efficiently than those previously studied. These are extremely encouraging and important findings. Hopefully these techniques will work just as well on humans!

The paper has just been published in the August issue of the Journal of Virology. It is highly technical, but obviously worth reading.