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Enhanced brain distribution of modified aspartoacylase

Canavan disease is a fatal neurological disorder caused by defects in the gene that produces the enzyme aspartoacylase. Enzyme replacement therapy can potentially be used to overcome these defects if a stable enzyme form can be produced that can gain access to the appropriate neural cells. Achieving...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab
Main Authors: Poddar, Nitesh K., Zano, Stephen, Natarajan, Reka, Yamamoto, Bryan, Viola, Ronald E.
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4252805/
https://ncbi.nlm.nih.gov/pubmed/25066302
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2014.07.002
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