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Article

Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy

1 May 2020

Abstract excerpt

Intravascular injection of certain adeno-associated virus vector serotypes can cross the blood-brain barrier to deliver a gene into the CNS. However, gene distribution has been much more limited within the brains of large animals compared to rodents, rendering this approach suboptimal for treatment of the global brain lesions present in most human neurogenetic diseases. The most commonly used serotype in animal...

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PMID: 32671406
DOI: 10.1093/brain/awaa161

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