Webinar – In vivo preclinical models for the study of neurodegenerative diseases

Date & Time	April 17, 11.00- 12.00
Address	Online
Location	Online (GotoWebinar)

Animal models are an indispensable research tool to understand, assess and treat neurodegenerative diseases in the preclinical setting. In this webinar, the speakers will highlight the contributions of discovery and experimentation in rodents and non-human primate (NHP) models to understand health and disease in humans and to preclinical drug development. The speakers will then focus on how to generate NHP models for neurodegenerative diseases and how to characterize them, using the example of a preclinical model of Tauopathy (viral overexpression) and the validation of a new gene therapy strategy for Parkinson’s disease (MPTP model).

Keywords: preclinical models, murine models, non-human primates, longitudinal assessment, Parkinson’s disease, tauopathies, intellectual disabilities, Down syndrome

Dr. Romina Aron Badin (NeurATRIS)

Dr. Romina Aron Badin received her PhD in gene therapy and brain imaging from the University College of London in 2005. She continued her research at the Institute of Child Health, in Great Ormond Street Hospital, London as a postdoctoral fellow working in gene therapy in rodent models of stroke. She then pursued her research in preclinical non-human primate models of brain disorders through a post-doctoral fellowship at the CEA focussing on Parkinson’s disease and new anti-dyskinetic treatments. She was recruited permanently in the CEA in 2008 and since then directs both the primate Neurosurgery and Behavioural platforms in MIRCen. She has been PI of over 30 projects involving non-human primates and has extensive technical expertise in the experimental design and execution of pre-clinical protocols for brain delivery of drugs, viral vectors and cells, as well as in the assessment of cognitive and motor function in primates. She has been a consultant for the MJFF, an expert in the SCHEER scientific committee commissioned by the EU, she is part of the steering committee of the GDR BioSimia that federates all primate laboratories in France, of the Basal Ganglia Club and the NECTAR (Network for European CNS Transplantation and Repair) board. Her current research interests include functional evaluation of innovative gene or cell therapies for Parkinson’s and Huntington’s diseases as well as the development of new primate models of Alzheimer’s and Parkinson’s diseases.

Dr. Marie-Christine Birling (Celphedia)

 

Dr. Marie-Christine Birling received her PhD in Molecular Biology Neurosciences from the University Strasbourg in 1994. She continued her research at the SmithKline Beecham, near London as a postdoctoral fellow trying to better understand the development of glial cells. She then pursued her research in finding new oligodendrocytes specific genes through a post-doctoral fellowship at the University of Edinburgh. She was recruited permanently at the Institut Clinique de la Souris in 2003. Since then she leads the genetic engineering team.  She advises scientists on the best approach for generating relevant animal models (mouse and rat). She has initiated more than 1500 à la carte models with some of them leaded to high impact scientific paper. Since 2013, she has implemented the CRISPR/Cas9 technology and now, most of the models are generated through this approach (Knock-Out, conditional Knock-Out, small Knock-In). Marie-Christine is also in charge of internal R&D projects. With her team, she has developed a new CRISPR approach (called CRISMERE) for the generation of structural genomic variants (here Down syndrome models, Birling et al., 2017).