Original title: AAV-mediated delivery in large animals
Authors: Blits, B. ; De Haan, M. ; Evers, M. ; Spronck, E. A. ; Motlík, Jan ; Bohuslavová, Božena ; Ellederová, Zdeňka ; Lewis, O. T. ; Johnson, D. ; Woolley, M. ; Gill, S. ; van Deventer, S. ; Konstantinová, P. ; Petry, H.
Document type: Papers
Conference/Event: Animal Models of Neurodegenerative Diseases /4./, Liblice (CZ), 20171022
Year: 2017
Language: eng
Abstract: Gene therapy is an attractive option for treatment of neurological diseases. Delivery of the therapeutic gene at the proper location is key for an effective treatment and remains challenging, especially in larger animals. For translation from smaller (rodents) to larger animals, dimensions are different, but also the immune system plays a more prominent role in larger animals. Direct intracranial parenchymal infusions usually result in local transduction of tissue, whereas intrathecal infusions result in a more widespread transduction in the brain. Depending on the indication, the desired expression pattern of the therapeutic gene is to be elucidated and is dictating the route of infusion.
Keywords: large animals
Project no.: LO1609 (CEP)
Funding provider: GA MŠk
Host item entry: The 4th Animal Models of Neurodegenerative Diseases

Institution: Institute of Animal Physiology and Genetics AS ČR (web)
Document availability information: Fulltext is available at the institute of the Academy of Sciences.
Original record: http://hdl.handle.net/11104/0280229

Permalink: http://www.nusl.cz/ntk/nusl-371497


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Research > Institutes ASCR > Institute of Animal Physiology and Genetics
Conference materials > Papers