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An Approach for Sustained Delivery of Therapeutic Agents to the Retina

Researchers at the University of Missouri - Columbia have invented a novel technology to treat retinal degenerative disorders. This approach will allow continuous delivery of therapeutic agents, such as bioactive proteins and peptides, to the retina over long periods of time after a single treatment. The researchers are performing preclinical studies treating the late-infantile form of the lysosomal storage disorder Neuronal Ceroid Lipofuscinosis (CLN2 disease) using a canine model. CLN2 disease results from mutations in the CLN2 gene. Dachshunds with a frameshift mutation in the canine ortholog of CLN2 develop a disorder very similar to human CLN2 disease. The human and canine disorders are characterized by progressive retinal degeneration resulting in blindness. Using the canine model, the researchers are evaluating the potential of this new technology for preventing retinal degeneration and loss of vision. The technology is also being investigated as a means of continuous delivery of therapeutic agents to the brain.

-Retinal Degenerative Disorders
-Age-Related Macular Degeneration
-Diabetic Retinopathy
-Neuronal Ceroid Lipofuscinoses (NCLs)
-Other lysosomal storage diseases
-Neurodegenerative diseases
-Other chronic disorders

-Cost Effective
-Reduced risk for scarring and inflammation associated with treatments requiring frequent injections or infusions
-Adult stem cell mediated approach

-Preclinical stage

Additional Details


University of Missouri

Intellectual Property Protection

Pending Patent

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