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Article

Long-Term Follow-Up After Gene Therapy for Canavan Disease

19 Dec 2012

Abstract excerpt

Canavan disease is a hereditary leukodystrophy caused by mutations in the aspartoacylase gene (ASPA), leading to loss of enzyme activity and increased concentrations of the substrate N-acetyl-aspartate (NAA) in the brain. Accumulation of NAA results in spongiform degeneration of white matter and severe impairment of psychomotor development. The goal of this prospective cohort study was to assess long-term safety...

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PMID: 23253610
DOI: 10.1126/scitranslmed.3003454

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