Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer

Abstract excerpt

<title>Abstract</title> <p>Lysosomal acid lipase deficiency (LAL-D) is an autosomal recessive disorder caused by mutations in the LIPA gene, which results in lipid accumulation leading to multi-organ failure. If left untreated, the severe form of LAL-D results in premature death within the first year of life due to failure to thrive and hepatic insufficiency. Enzyme replacement therapy is the only available suppo...

Topics

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• Adaptive Immunity
• Disease Models, Animal
• Gene Transfer Techniques
• Genetic Therapy
• Genetic Vectors
• Immunity, Innate
• Lysosomal Storage Diseases
• Mutation

Identifiers and source

Literature Corpus work

9e664b55-e289-5c6c-be08-2d980314f666

DOI

10.21203/rs.3.rs-4354172/v1