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Intrathecal enzyme replacement therapy: Successful treatment of brain disease via the cerebrospinal fluid

Treatment of brain disease with recombinant proteins is difficult due to the blood-brain barrier. As an alternative to direct injections into the brain, we studied whether application of high concentrations of therapeutic enzymes via intrathecal (IT) injections could successfully drive uptake across...

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Detalhes bibliográficos
Main Authors: Dickson, Patricia, McEntee, Michael, Vogler, Carole, Le, Steven, Levy, Beth, Peinovich, Maryn, Hanson, Stephen, Passage, Merry, Kakkis, Emil
Formato: Artigo
Idioma:Inglês
Publicado em: 2007
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3009387/
https://ncbi.nlm.nih.gov/pubmed/17321776
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2006.12.012
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