Perinatal systemic gene delivery using adeno-associated viral vectors

Neurodegenerative monogenic diseases often affect tissues and organs beyond the nervous system. An effective treatment would require a systemic approach. The intravenous administration of novel therapies is ideal but is hampered by the inability of such drugs to cross the blood–brain barrier (BBB) a...

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Dades bibliogràfiques
Publicat a:Front Mol Neurosci
Autors principals: Karda, Rajvinder, Buckley, Suzanne M. K., Mattar, Citra N., Ng, Joanne, Massaro, Giulia, Hughes, Michael P., Kurian, Manju A., Baruteau, Julien, Gissen, Paul, Chan, Jerry K. Y., Bacchelli, Chiara, Waddington, Simon N., Rahim, Ahad A.
Format: Artigo
Idioma:Inglês
Publicat: Frontiers Media S.A. 2014
Matèries:
Neuroscience
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4231876/
https://ncbi.nlm.nih.gov/pubmed/25452713
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fnmol.2014.00089
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