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-acetylaspartate (NAA) in the brain. Accumulation of NAA results in spongiform degeneration of white matter and seve...

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Detalhes bibliográficos
Main Authors: Leone, Paola, Shera, David, McPhee, Scott W.J., Francis, Jeremy S., Kolodny, Edwin H., Bilaniuk, Larissa T., Wang, Dah-Jyuu, Assadi, Mitra, Goldfarb, Olga, Goldman, H. Warren, Freese, Andrew, Young, Deborah, During, Matthew J., Samulski, R. Jude, Janson, Christopher G.
Formato: Artigo
Idioma:Inglês
Publicado em: 2012
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3794457/
https://ncbi.nlm.nih.gov/pubmed/23253610
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3003454
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