Article

Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease

2019-06-19

Abstract excerpt

The neuronal ceroid lipofuscinoses (NCLs), more commonly referred to as Batten disease, are a group of inherited lysosomal storage disorders that present with neurodegeneration, loss of vision and premature death. There are at least 13 genetically distinct forms of NCL. Enzyme replacement therapies and preclinical studies on gene supplementation have shown promising results for NCLs caused by lysosomal enzyme defi...

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Literature Corpus work: a7ce1fe2-d31d-5d15-86ab-697baf5e4979
DOI: 10.1101/673848

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