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Project Title:
Development of an Enzyme Replacement Therapy for Primary Congenital Glaucoma
Principle Investigator:
Rachida Bouhennni, PhD
Institution:
Akron Children’s Hospital
Enzyme Injection
The Cure Glaucoma Foundation funded this project to test injecting an enzyme into the eye of a glaucoma mouse model.
The investigators delivered the enzyme by injecting it into the front part of the eye. Analysis confirmed the distribution of the enzyme in the front of the eye. Future studies will investigate the effect the enzyme could have on the eye after injection.
This project’s results give hope that therapeutic proteins can treat eye diseases caused by missing or deficient proteins.
For example, Cytochrome p450 1b1 deficiency causes Primary Congenital Glaucoma. Researchers can use therapeutic proteins to inhibit or stimulate biological processes, like regulating eye fluid production and intraocular pressure. Scientists can produce these proteins in the laboratory using proper techniques. These techniques allow production of biologically active, correctly shaped proteins and purification of large quantities at reasonable cost.
Protein Therapy
Protein therapy provides a safer, and more permanent approach to treat diseases compared to other approaches for replacing deficient enzymes such as bone marrow transplantation, cell transplantation or gene therapy. Although researchers are widely exploring gene therapy in glaucoma and it shows promise, protein therapy may offer many advantages. Protein therapy uses well-defined, precisely structured molecules, delivers optimal protein doses, and produces predictable biological effects. Furthermore, with protein therapy there is no need for viral carriers that increase the risk for inflammatory responses, toxicity, and cancer issues.