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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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Detalles Bibliográficos
Autores principales: von Jonquieres, Georg, Fröhlich, Dominik, Klugmann, Claudia B., Wen, Xin, Harasta, Anne E., Ramkumar, Roshini, Spencer, Ziggy H. T., Housley, Gary D., Klugmann, Matthias
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4763065/
https://www.ncbi.nlm.nih.gov/pubmed/26941604
http://dx.doi.org/10.3389/fnmol.2016.00013