Perinatal systemic gene delivery using adeno-associated viral vectors

Perinatal systemic gene delivery using adeno-associated viral vectors

Neurodegenerative monogenic diseases can also affect a broad range of tissues and organs throughout the body. 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 b...

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Bibliographic Details
Main Authors: Rajvinder eKarda, Suzanne M K Buckley, Citra N Mattar, Joanne eNg, Giulia eMassaro, Michael eHughes, Manju A Kurian, Julien eBaruteau, Paul eGissen, Jerry K Y Chan, Chiara eBacchelli, Simon N Waddington, Ahad A Rahim
Format: Article
Language:English
Published: Frontiers Media S.A. 2014-11-01
Series:Frontiers in Molecular Neuroscience
Subjects:
Gene Therapy
Metabolic Diseases
neurodegenerative disease
intravenous
mouse models
non-human primates
Online Access:http://journal.frontiersin.org/Journal/10.3389/fnmol.2014.00089/full

Internet

http://journal.frontiersin.org/Journal/10.3389/fnmol.2014.00089/full

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