Long-Term Follow-Up After Gene Therapy for Canavan Disease
نویسندگان
چکیده
منابع مشابه
Long-term follow-up after gene therapy for canavan disease.
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 asses...
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ژورنال
عنوان ژورنال: Science Translational Medicine
سال: 2012
ISSN: 1946-6234,1946-6242
DOI: 10.1126/scitranslmed.3003454