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Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease

Samantha J. Murray, Katharina N. Russell, Tracy R. Melzer, Steven J. Gray, Stephen J. Heap, David N. Palmer, Nadia L. Mitchell

2021Experimental Eye Research27 citationsDOI

Topics & Concepts

Batten diseaseRetinal degenerationRetinalElectroretinographyNeuronal ceroid lipofuscinosisRetinaErgAtrophyMedicineOphthalmologyLipofuscinPathologyDiseaseBiologyNeuroscienceLysosomal Storage Disorders ResearchRetinal Development and DisordersRetinal Diseases and Treatments
Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease | Litcius