Article

A blood-brain-barrier penetrant AAV gene therapy rescues neurological deficits in mucolipidosis IV mice

2023-11-05

Abstract excerpt

Mucolipidosis IV (MLIV) is a rare, autosomal recessive, lysosomal disease characterized by intellectual disability, motor deficits and progressive vision loss. Using AAV9 and AAV-PHP.B as delivery vectors, we previously demonstrated the feasibility of modifying disease course in a mouse model of MLIV by the human MCOLN1 gene transfer. Here, using a primate-enabling capsid AAV.CPP.16 (CPP16), we constructed a new,...

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Literature Corpus work: 0ae68e0f-31a7-546a-a50e-f830dab3b8a3
DOI: 10.1101/2023.11.03.565568

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