Webinar
α-Synuclein and Organelles: Key Players in the Neurodegeneration Puzzle
About the webinar – α-Synuclein and Organelles: Key Players in the Neurodegeneration Puzzle
Cellular homeostasis, regulation of oxidative stress and autophagy are processes finely regulated by specialized organellar structures, such as lysosomes and mitochondria. Emerging evidence shows that neurodegenerative diseases such as Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis are characterized by dysfunction in these critical organelles. Moreover, in all these pathologies protein aggregation is evident to be a common hallmark, appointing these disorders as proteinopathies.
To explore the interplay between organellar functions and proteinopathies, we create in vitro disease models by means of iCell® DopaNeurons, and SH-SY5Y neuroblastoma cells triggered by appropriate stressors.
Pathological phenotypes were investigated by high content imaging and electrophysiology, using single organellar-based analysis. In parallel, to increase complexity and relevance of our model, we included co-culture approaches and Cell Painting imaging, expanding the powerfulness of our platform.
Results showed that both SH-SY5Y and DopaNeurons phenotypes well recapitulate pathological hallmarks and distinct ionic currents were recorded from lysosomal patches isolated from both healthy and mutated cells.
These results confirm that the platform we established is suitable for modeling proteinopathies and organellar involvement using relevant iPSC-derived models in a scalable manner. This integrated system enables miniaturized drug screening for identification of novel therapeutic targets in neurodegenerative diseases.
In this webinar you will learn about:
The role of lysosomal and mitochondrial dysfunction in neurodegenerative diseases
How iPSC-derived neuronal models can be used to reproduce and study disease-relevant phenotypes
How high-content imaging and organelle electrophysiology can help uncover new therapeutic targets.
Keywords
Neurodegenerative Diseases; Organelles; iPSC-derived Neurons; Drug Discovery; High Content Imaging; Parkinson’s Disease; α-synuclein; TMEM175; Cell Painting; Drug Discovery; Target ID; Screening Platform; Cell-based assays
Meet the speakers
Elisa Tinelli – Senior Program Manager, Golgi Neurosciences
Donatella Bardelli – Principal Scientist Advanced and Cellular Assays, Axxam
Elisa Tinelli graduated in Pharmaceutical Biotechnology at the University of Milan (Italy) and earned a joint PhD in Cellular and Molecular Biology (San Raffaele University, Italy/Open University, UK). She completed a postdoc at ETH Zurich, investigating molecular mechanisms of neuromuscular disorders and peripheral neuropathies. Then, she joined UCL London where she focused on the identification of novel treatment for Neuronal Ceroid Lipofuscinosis, while serving as Risk Manager for a multicentric European consortium. Before joining Golgi Neurosciences, Elisa was Principal Investigator in the Discovery Research department at Axxam. Currently, as a Senior Program Manager in Golgi Neurosciences, Elisa focuses on accelerating therapeutic development for neurodegenerative and rare diseases.
Donatella Bardelli is a Principal Scientist in the Advanced and Cellular Assays Group at Axxam, with more than five years of experience working with iPSC-derived cellular models.
Her research focuses on the development of iPSC-based assays designed for scalability and miniaturization, enabling their application in high-throughput screening for drug discovery. At Axxam, she has successfully developed several advanced cellular assays using human induced pluripotent stem cell (iPSC) models.
Donatella holds a Master’s Degree in Biology and Biomedical Applications and a PhD in Molecular and Translational Medicine. She has a strong background in cell biology and is the author and co-author of several scientific publications in the field.