Grant: Project Grant

Glaucoma is considered the leading cause of irreversible blindness worldwide. The increased intraocular pressure (IOP) has been established as the main risk factor for the development of glaucoma. Unfortunately, currently approved therapies for glaucoma that target IOP are unable to prevent the detrimental course of the disease, raising the need for novel treatments that can prevent or stop the progression in glaucoma. A family of enzymes that degrade proteins, called proteases, are activated in glaucoma. Abnormal activation of these proteases in glaucoma can cause inflammation by releasing harmful molecules in the eye. Recently, we have developed a special protein that replaces those in the cell so that instead of harmful factors, special therapeutic factors are released. The long term goal of this novel and first-in-class strategy is to have the retinal cells release the special, therapeutic proteins during the first signs of glaucomatous damage in the eye. Our proposed "inducible" therapeutics will be positioned in retinal cells in advance of disease, and will work as needed to reduce the negative systemic and off-target effects frequently associated with current therapies for eye diseases. The know-how gained from these studies can lead to development of novel gene therapies for treatment of other brain diseases such traumatic brain injury, multiple sclerosis, Alzheimer's and Parkinson's disease.