AAV-mediated gene delivery attenuates neuroinflammation in feline Sandhoff disease

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• 作者：Allison M. Bradbury^a ; ^b ; ^&dagger ; ; ^{brada@upenn.edu} ; Tiffany A. Peterson^a ; ^&Dagger ; ; Amanda L. Gross^a ; Stephen Z. Wells^c ; Victoria J. McCurdy^a ; ^b ; ^§ ; ; Karen G. Wolfe^b ; John C. Dennis^b ; Brandon L. Brunson^b ; Heather Gray-Edwards^a ; Ashley N. Randle^a ; Aime K. Johnson^c ; Edward E. Morrison^b ; Nancy R. Cox^a ; ^d ; Henry J. Baker^a ; Miguel Sena-Esteves^e ; Douglas R. Martin^a ; ^b
• 关键词：AAV ; adeno-associated virus ; CNS ; central nervous system ; DCN ; deep cerebellar nuclei ; GFAP ; glial fibrillary acidic protein ; Hex ; β-N-acetylhexosaminidase ; Iba1 ; ionized calcium-binding protein 1 ; ICV ; intracerebroventricular ; IL-1β ; interleukin 1 beta ; MHC-II ; major histocompatibility complex II ; MIP-1α ; macrophage inflammatory protein-1 alpha ; PBMC ; peripheral blood mononuclear cell ; SD ; Sandhoff disease ; Sphk1 ; sphingosine kinase 1 ; TGFβ1 ; transforming growth factor beta 1 ; TNF-α ; tumor necr
• 刊名：Neuroscience
• 出版年：2017
• 出版时间：6 January 2017
• 年：2017
• 卷：340
• 期：Complete
• 页码：117-125
• 全文大小：2922 K
• 卷排序：340

文摘

Neuroinflammation is a prominent pathological feature of feline Sandhoff disease. A robust infiltration and activation of microglia were evident in CNS of SD cats. MHCII and MIP-1α were up-regulated in the brain of SD cats. Intracranial AAV gene therapy attenuated inflammation, normalizing MHCII and MIP-1α.