Glaucoma is a chronic, neurodegenerative disease that is associated with damage to retinal ganglion cells and central parts of the visual pathway. Untreated glaucoma can lead to total and irreversible blindness. The current glaucoma therapy is based primarily on various methods of lowering intraocular pressure. However, the progression of vision loss continues to persist in some patients despite the use of maximum doses of intraocular pressure lowering medications. Nowadays, there are no neuroprotective   herapies with proven efficacy for glaucoma. For this reason, the search for such therapies to inhibit retinal ganglion cell death and stimulate their regeneration is an important and current problem.

The aim of our project is to develop an innovative experimental gene therapy for the neuroprotection of retinal ganglion cells in glaucoma. Based on our previous observations, as a target of our therapy we have chosen the RNA-binding protein HuR. This protein is crucial element in regulating the response of cells to damaging factors, including elevated intraocular pressure. Under experimental conditions, in experimental glaucoma in animals as well as in glaucoma patients we showed that the HuR protein content in the retinal neurons is significantly lowered. Therefore, we plan to experimentally increase the HuR protein content in retinal ganglion cells by gene therapy approach in experimentally induced glaucoma in rats associated with high intraocular pressure.

From: Smedowski A. et al. Increased intraocular pressure alters the cellular distribution of HuR protein in retinal ganglion cells - A possible sign of endogenous neuroprotection failure. Biochim Biophys Acta Mol Basis Dis. 2018 Jan;1864(1):296-306.