Gene Therapy Associated With Vision Preservation in Retinal Disease - AJMC.com Managed Markets Network

“We uncovered evidence for the first time that CaMKII is a key regulator of the survival of retinal ganglion cells in both normal and diseased retinas and could be a desirable therapeutic target for vision preservation in conditions that damage the axons and somas of retinal ganglion cells,” senior author Bo Chen, PhD, associate professor of ophthalmology and neuroscience, and director of the Ocular Stem Cell Program at the Icahn School of Medicine at Mount Sinai, said in a statement.

In their study, the investigators implemented a gene therapy approach through which they demonstrated how reactivation of CaMKII and its downstream signaling in targeted retinal ganglion cells could provide robust protection against the progression of vision loss or impairment in various disease and injury models.

After testing the enzyme across the range of injury and disease animal models, the researchers found that the survival of retinal ganglion cells across many pathologies could be regulated by CaMKII.

However, exposure of retinal ganglion cells to toxins or trauma from a crush injury to the optic nerve compromised pathway signaling by CaMKII.

The research suggested a correlation between CaMKII activity and retinal ganglion cell survival, which led the researchers to question whether activating the CaMKII pathway would offer protective qualities to the retinal ganglion cells.

Through methods of gene therapy, they mutated an amino acid within CaMKII to create a more active version of the enzyme, then introduced it into original retinal ganglion cells to boost activity levels.

According to cell activity measured by electroretinogram and patterns of activity in the visual cortex, higher retinal ganglion cell survival rates were associated with greater changes of preserved visual function.