Rare disease (RD) is one area where national and international multidisciplinary collaborations are valuable to overcome some of the present challenges in research. Access to infrastructures, collaborative research and policy platforms, virtual platforms for data integration are some examples of means to overcome bottlenecks for diagnosis and treatment of RD patients. One of such infrastructures are biobanks which are important drivers for creating scientific knowledge by collecting, storing and distributing human biological samples and associated data with quality assurance management systems and following ethical and legal guidelines.
Biological samples can be used as valuable starting materials for generating cellular models of disease. This can occur, for example, by generating induced pluripotent stem cell (iPSC) lines from cells and tissues, and then differentiating the iPSC lines into specific cell types for disease modelling or drug screening. Advantageously, iPSC lines can be generated from any individual, are a renewable cell resource, and can be used to produce cell types that are difficult to access or obtain from native tissues (e.g. specialized brain cell types). In the present biobank training, the aim is to provide an overview on how human pluripotent stem cells can be used as research tools.
Dr. Benjamin Schmid will give a 20 min presentation on Gene editing approaches overview during day 2 (October 26th) at 11:00.
The training is aimed at biomedical researchers, medical professionals, and biobank managers who want to learn about the application of human pluripotent stem cell (hPSC) lines in rare disease research. Practical considerations from start to finish will be presented, including the sourcing of biological material for pluripotent stem cell generation, making pluripotent stem cell lines, hPSC cell line banking, data management, and methods for genetically engineering and differentiating hPSC lines. We will address the topic from diverse points of view, including representatives from biomedical ethics, patient organizations, applied research and industry.
Register here before September 27th.