Recombinant human myelin-associated glycoprotein promoter drives selective AAV-mediated transgene expression in oligodendrocytes

Recombinant human myelin-associated glycoprotein promoter drives selective AAV-mediated transgene expression in oligodendrocytes

Leukodystrophies are hereditary central white matter disorders caused by oligodendrocyte dysfunction. Recent clinical trials for some of these devastating neurological conditions have employed an ex vivo gene therapy approach that showed improved endpoints because cross-correction of affected myelin...

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Bibliographic Details
Main Authors: Georg evon Jonquieres, Claudia Bettina Klugmann, Dominik eFröhlich, Xin eWen, Anne Editha Harasta, Roshini eRamkumar, Ziggy HT Spencer, Gary D Housley, Matthias eKlugmann
Format: Article
Language:English
Published: Frontiers Media S.A. 2016-02-01
Series:Frontiers in Molecular Neuroscience
Subjects:
Gene Therapy
Myelin-Associated Glycoprotein
Oligodendroglia
aav
white matter disorders
Leukodystrophy
Online Access:http://journal.frontiersin.org/Journal/10.3389/fnmol.2016.00013/full

Internet

http://journal.frontiersin.org/Journal/10.3389/fnmol.2016.00013/full

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