Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

Enzyme replacement therapy has revolutionized the treatment of the somatic manifestations of lysosomal storage diseases (LSD), although it has been ineffective in treating central nervous system (CNS) manifestations of these disorders. The development of neurotrophic vectors based on novel serotypes...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Hinderer, Christian, Bell, Peter, Gurda, Brittney L, Wang, Qiang, Louboutin, Jean-Pierre, Zhu, Yanqing, Bagel, Jessica, O'Donnell, Patricia, Sikora, Tracey, Ruane, Therese, Wang, Ping, Haskins, Mark E, Wilson, James M
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4429692/
https://ncbi.nlm.nih.gov/pubmed/25027660
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.135
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