AAV-mediated delivery in large animals

0485155 - ÚŽFG 2018 CZ eng K - Conference Paper (Czech conference)
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.
AAV-mediated delivery in large animals.
The 4th Animal Models of Neurodegenerative Diseases. Liběchov: ÚŽFG AV ČR, v. v. i., 2017, s. 19-20.
[Animal Models of Neurodegenerative Diseases /4./. Liblice (CZ), 22.10.2017-24.10.2017]
R&D Projects: GA MŠMT(CZ) LO1609
Institutional support: RVO:67985904
Keywords : large animals
OECD category: Neurosciences (including psychophysiology

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.
Permanent Link: http://hdl.handle.net/11104/0280229