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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Bibliographic Details
Published in:Front Mol Neurosci
Main Authors: 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
Language:Inglês
Published: Frontiers Media S.A. 2014
Subjects:
Neuroscience
Online Access: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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