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We aim at understanding how the physiology of the central nervous system (CNS) becomes impaired in neurodegenerative diseases. Etiology and pathology of ageing-related disorders like Parkinson´s (PD) and Alzheimer´s diseases (AD) are still poorly understood, making it difficult to develop therapeutic strategies. Such strategies, however, are in desperate demand in the ageing societies of the western world.
We make use of highly efficient viral vector systems to introduce genetic information into selected CNS target cell populations or to knock-down expression of proteins of interest by RNAi. For example, we generated novel animal models of AD and PD by over-expression of aSynuclein, Tau, APP and their disease-relevant mutants in the rodent brain by adeno-associated viral (AAV) vectors, making it possible to study mechanisms of neurodegeneration not accessible in conventional transgenic mice.
In order to have a more detailed view on neuropathology in living neurons, we express fluorescent sensor proteins in models of neurodegeneration, revealing sub-cellular events impairing neuronal functions well before the cell is prone to degeneration.
Importantly, gene transfer by AAV vectors is effective not only in rodents but also in the primate and even the songbird brain, and can be accomplished in cultured primary brain cells, in organotypic slice cultures as well as in any brain area of interest.
Another pillar of our research lies in the use of viral vectors as gene therapy tools, e.g. for treatment of monogenic disorders, and in technical developments like cell type specific and regulated transgene expression in the brain of rodents and primates.
We make use of highly efficient viral vector systems to introduce genetic information into selected CNS target cell populations or to knock-down expression of proteins of interest by RNAi. For example, we generated novel animal models of AD and PD by over-expression of aSynuclein, Tau, APP and their disease-relevant mutants in the rodent brain by adeno-associated viral (AAV) vectors, making it possible to study mechanisms of neurodegeneration not accessible in conventional transgenic mice.
In order to have a more detailed view on neuropathology in living neurons, we express fluorescent sensor proteins in models of neurodegeneration, revealing sub-cellular events impairing neuronal functions well before the cell is prone to degeneration.
Importantly, gene transfer by AAV vectors is effective not only in rodents but also in the primate and even the songbird brain, and can be accomplished in cultured primary brain cells, in organotypic slice cultures as well as in any brain area of interest.
Another pillar of our research lies in the use of viral vectors as gene therapy tools, e.g. for treatment of monogenic disorders, and in technical developments like cell type specific and regulated transgene expression in the brain of rodents and primates.
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Carolin Knauer, Henrike Haltern,Eric Schoger,Sebastian Kuegler, Lennart Roos,Laura C. Zelarayan,Gerd Hasenfuss,Wolfram-Hubertus Zimmermann,Bernd Wollnik,Lukas Cyganek
MOLECULAR THERAPY NUCLEIC ACIDSno. 1 (2024): 102123-102123
Pretty Garg, Franziska Würtz, Fabian Hobbie, Klemens Buttgereit,Abhishek Aich,Kristian Leite,Peter Rehling,Sebastian Kügler,Mathias Bähr
Journal of Neuroinflammationno. 1 (2024): 1-16
Nina Wittenmayer, Andonia Petkova-Tuffy, Maximilian Borgmeyer, Chungku Lee,Juergen Becker, Andreas Boening,Sebastian Kuegler,Jeongseop Rhee,Julio S. Viotti,Thomas Dresbach
Frontiers in Cellular Neuroscience (2023): 1182493-1182493
Human Molecular Geneticsno. 3-4 (2021): 247-264
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