Efficacy of Dual Intracerebroventricular and Intravitreal CLN5 Gene Therapy in Sheep Prompts the First Clinical Trial to Treat CLN5 Batten Disease
FRONTIERS IN PHARMACOLOGY（2023）
关键词
neuronal ceroid lipofuscinosis,neurodegenerative disease,gene therapy,adeno-associated virus,intracerebroventricular,intravitreal,sheep
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