New model systems enable easy culturing of patient cells in the lab for functional studies, which can be highly useful for drug development and personalized medicine. For instance, intestinal and airway organoid culture methods can be used to study cystic fibrosis (CF), a rare monogenic disease that is caused by aberrant function of an ion channel termed CFTR. These new organoid methodologies are particularly interesting as they resemble the native tissue organization, can be grown in large quantities and enable storage in biobanks.
In the second BIH Multi-Omics Lecture on 15 October 2020, Jeffrey Beekman, Full Professor at the University Medical Center (UMC) Utrecht, who pioneered the use of intestinal and airway organoid culture methods to study CF, will discuss how current therapies for CF that aim to restore CFTR function are revolutionizing the treatment of CF, causing unprecedented effects in people with CF. The presentation will provide an example for the use of patient-derived cell systems for studies of rare, genetic disease in the context of basic science, drug development, individual disease development and treatment thereof.
About the Speaker
Dr. Jeffrey Beekman is full professor of Cellular Disease Models at the University Medical Center (UMC) Utrecht in the Netherlands. He guides a translational research group that focuses on the development and validation of patient-derived cell models for basic science studies, drug development and individualized medicine applications. He has been trained in molecular and cellular biology within an immunological context, and became principle investigator in 2010, focusing on CF. He has pioneered the use of 3D stem cell models for studies of CF and developed functional assays that were used to study CF Transmembrane Conductance Regulator (CFTR) function and CFTR-directed therapies. His work was awarded with various international prizes including the ECFS award 2017 and the European Respiratory Society Excellence award for research in CF 2017 and the Prix Galien 2019. Currently, he aims to develop new disease models for various monogenic diseases (e.g., primary ciliary dyskinesia, microvillus inclusion disease) and viral airway infections such as SARS-CoV-2.
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