Intravitreal enzyme replacement inhibits progression of retinal degeneration in canine CLN2 neuronal ceroid lipofuscinosis
Author:
Funder
U.S. National Institutes of Health
Publisher
Elsevier BV
Subject
Cellular and Molecular Neuroscience,Sensory Systems,Ophthalmology
Reference31 articles.
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1. Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study;The Lancet Neurology;2024-01
2. Intravitreal gene therapy preserves retinal function in a canine model of CLN2 neuronal ceroid lipofuscinosis;Experimental Eye Research;2023-01
3. Strategies to treat neurodegeneration in neuronal ceroid lipofuscinosis: a view onto the retina;Neural Regeneration Research;2023
4. Inborn Errors of Metabolism in Dogs: Historical, Metabolic, Genetic, and Clinical Aspects;Topics in Companion Animal Medicine;2022-11
5. Ongoing retinal degeneration despite intraventricular enzyme replacement therapy with cerliponase alfa in late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease);British Journal of Ophthalmology;2022-06-30
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