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: von Jonquieres, Georg, Fröhlich, Dominik, Klugmann, Claudia B., Wen, Xin, Harasta, Anne E., Ramkumar, Roshini, Spencer, Ziggy H. T., Housley, Gary D., Klugmann, Matthias
Format: Online
Language:English
Published: Frontiers Media S.A. 2016
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4763065/